376 Adeno-associated Viral Vector (AAV) Companies - Worldwide

2A Pharma is a clinical stage Swedish/Danish biopharmaceuticals company focused on discovering, developing and commercialising novel, cost effective prophylactic and therapeutic vaccines based on our patented vaccine platform. Our lead vaccine candidate, 2AP01, is a HPV (human papillomavirus) vaccine formulated without adjuvant. We believe 2AP01 will offer significant advantages over existing vaccines including broader protection against more HPV types and lower production cost. Our vaccine technology is based on adeno-associated virus-like particles (AAVLPs), that assemble viral capsids without containing viral genomes; hence they are replicative-defective and non-pathogenic. The viral capsid has repetitive immunogenic sites that can be modified to encompass antigenic epitopes for a protein of interest, generating an immune response where both the innate and the adaptive immune system are activated. In other words, 2A Pharma exploit the efficacy of the individuals’ own immune system to generate AAVLP-based vaccines that, without the addition of adjuvants, are able to develop highly effective antibodies with selected specificities and functions. The technology enables the production of highly efficient vaccines with significant competitive advantages

4basebio is enabling next generation cell and gene therapies and vaccines with its technologies and solutions. We are able to design, manufacture and supply application-specific synthetic DNA or mRNA as well as targeted non-viral vectors for the delivery of nucleic acid payloads. Our novel synthetic DNA technology offers unique customisation potential, rapid turn-around times, and improved safety profiles, addressing the current limitations of plasmids and other DNA technologies. We currently offer four DNA construct types, each ideally suited for use in viral and non-viral vector applications, genome editing, vaccines and therapeutics, and DNA vaccine applications, respectively. Our proprietary non-viral delivery system, Hermes™, is a nanoparticle vector that can be customised to target cells or tissues of interest for a range of applications. This combines the safety and efficiency of lipid-based nanoparticles (LNPs) with the specificity of a targeting system, addressing the shortcoming associated with both viral vectors and non-targeted LNPs.

4DMT is a clinical stage biopharma company inventing and developing innovative products to unlock the full potential of genetic medicine to treat large market and rare diseases. Our vector discovery platform Therapeutic Vector Evolution harnesses the power of directed evolution, a Nobel Prize-winning technology, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors with improved therapeutic profiles compared to wild-type vectors. Our product design, development and manufacturing engine empowers us to efficiently create our valuable and diverse product pipeline. This combination of bold innovation and relentless execution gives 4DMT the capability to revolutionize genetic medicines and to strive for potential curative therapies. We have built a deep portfolio of genetic medicine product candidates, with five product candidates in clinical trials for seven patient populations. We are taking genetic medicine to new heights and into new disease areas to overcome challenges not addressed by existing medicines. This incredible challenge drives our relentless and talented team. Our people are the essence of our inspiring, innovative and collaborative environment that encourages individual contributions and provides opportunities for career development. We seek the best in the industry who identify with our mission and our bold and innovative results-driven approach. Our 4 Guiding Principles drive the way we work together: - Dare to Cure - Break Boundaries - Beyond Yourself - Prepare & Execute Relentlessly Join us in creating the future of genetic medicine.

AaviGen GmbH is a biotechnology start-up located in Heidelberg, Germany. Our primary purpose is the development and commercialization of precision gene therapies for cardiovascular and cardiopulmonary diseases. Our business model builds on the success of the InoCard GmbH, a predecessor that was acquired by uniQure N.V. and subsequently entered into a development alliance with Bristol-Myers-Squibb. Please find our imprint ("Impressum") and data privavy statement ("Datenschutzerklärung") at https://aavigen.com/content/imprint.html and https://aavigen.com/content/privacy.html

AAVnerGene Inc is an innovative company specialized in AAV technologies. It is founded by a group of AAV gene therapy enthusiasts who dream to serve the community, promote and lead the future of gene therapy. Our passion is to solve the bottleneck problems in AAV gene therapy and find more and better cures for all the patients. We believe that hard core technologies are the key to achieve it. With 20 years of frontline gene therapy experience, our team develops novel AAV vectors, methods and strategies that can efficiently produce, deliver and express high quality vectors into specific cells. Our goal is to increase AAV gene therapy efficacy and decrease the cost, making it accessible and affordable to patients. Our highly complex AAV capsid libraries allow us to efficiently select the best AAV capsid for each cell type in a high-throughput manner.

Aavocyte, Inc. is a biotech company registered in the State of Washington in 2019. Aavocyte specializes in developing various precise targeting T cell immunotherapy products to treat solid tumors.

Our mission is to make you stronger. The company was founded by a family directly impacted by three muscle wasting diseases: Duchenne muscular dystrophy, cancer cachexia and COPD. Our fight is personal and our commitment to developing better and more effective therapies is undaunted. AVGN7 prevents muscle wasting in different animal models and can restore muscle mass and strength even after wasting has already occurred. It works when injected directly into muscle or when administered systemically to all muscles. It was also designed to avoid the potentially serious side-effects of other "myostatin attenuating" therapies. AVGN7 for the durable maintenance of muscle mass and function.

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases.

Driven by a bold founding vision: Empowering patients from within, Abintus is pioneering potentially curative, off-the-shelf genetic medicines that engineer cells directly inside the patient’s body, also termed in vivo. These therapies are based on Abintus' clinically proven Modular Delivery Platform (MDP). The company’s two initial, fit-for-purpose, In Vivo programs are: 1. Highly selective gene expression targeting cell type/state via synthetic biology (e.g., In Vivo CAR-NK/T/Treg, In Vivo TCR-T) with applications across multiple gene delivery systems (e.g., AAV, lenti, adeno, retro) and therapeutic areas including leukemia, solid tumors, autoimmune, infectious, and genetic disease. 2. Simultaneous engineering of a diverse repertoire of active innate and adaptive immune cells (e.g., In Vivo CAR-X) with applications in diseases such as lymphoma and solid tumors. Abintus is leveraging over 20 years and $275 million of prior technology advancement for in vivo engineering including the treatment of over 350 patients through Phase 3 registration trials. Our company name comes from the Latin words “ab intus”, which means “from within”. We chose this name for 3 reasons: 1) Patients: We are pursuing product candidates that are designed to directly empower patients’ own immune cells to fight their disease from within. 2) Science: Our team is committed to scientific excellence and continual learning. Our differentiated technologies have the potential to directly reprogram activated immune cells “in situ”, or from within. 3) Culture: We believe our ability to achieve our vision will come from within - by building a performance-driven culture that prioritizes career development and rewards outstanding contributions. Abintus’ initial funding syndicate included Takeda Ventures and The Leukemia & Lymphoma Society’s Therapy Acceleration Program® (LLS TAP). Abintus Bio, Inc. (“Abintus”) is headquartered in San Diego. www.abintusbio.com.

Acepodia is a clinical-stage biotechnology company developing innovative and highly effective cell therapies for cancer that are broadly accessible for patients. Leveraging its Antibody-Cell Conjugation (ACC) technology, the company links tumor-targeting antibodies to its proprietary immune effector cells, such as gamma delta T cells and natural killer (NK) cells to create novel antibody cell effector (ACE) therapies, which have increased binding strength against tumors that express low levels of tumor antigens. Acepodia is made up of seasoned leadership and scientific experts dedicated to advancing its robust pipeline of off-the-shelf ACE therapies with the potential to bring powerful, accessible cell therapies to a broad population of patients across a variety of solid tumors and hematologic cancers.

Adrenas Therapeutics is a company that was created with a single mission: to work with scientists, physicians, and patients in developing a gene therapy for people affected by CAH. Adrenas is a subsidiary of BridgeBio, a public company dedicated to finding, developing, and delivering breakthrough medicines for genetic diseases to patients as quickly and safely as possible.

Adverum Biotechnologies, Inc., a clinical-stage gene therapy company, engages in developing gene therapy product candidates to treat ocular and rare diseases. Its pipeline of product candidates includes ADVM-022, an adeno- associated virus (AAV). 7m8-aflibercept for the treatment of wet age-related macular degeneration; ADVM-043, an investigational gene therapy candidate for the treatment of alpha-1 antitrypsin deficiency; and ADVM-053, a preclinical gene therapy product candidate for the treatment of hereditary angioedema. The company has collaboration agreements with Editas Medicine, Inc. to leverage its proprietary AAV vectors for genome editing technologies to treat up to five inherited retinal diseases; and Regeneron Pharmaceuticals, Inc. for the development of up to eight distinct ocular therapeutic targets. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was founded in 2006 and is headquartered in Redwood City, California.

Adze Biotechnology is a biotechnology company that develops a platform of immunotherapies to treat solid tumors and enhance anti-tumor immunity.

Affinia Therapeutics is an innovative gene therapy company focused on broadening the reach of gene therapy. Our proprietary Affinia Rationally-designed Therapies (ART) platform is created to address key limitations of conventional gene therapies, by developing novel capsids, novel promoters, and novel manufacturing approaches to developing gene therapies for rare and prevalent devastating diseases. We are backed by a strong syndicate of life science investors and have ambitious plans to have a dramatic impact on the lives of patients around the world.

Agathos Biologics is a biotechnology company pursuing transformational science in biomanufacturing, biologic payload delivery, and cell and gene therapy. Discoveries in bioprocessing and genetic characterization and control have created an abundance of scientific possibilities that can help us all lead better lives. Our mission as the good science company is to create breakthrough products and services within a strong ethical and moral framework that benefit everyone. We believe in science that serves and have a relentless focus on serving our clients, employees, and society. For more information, please visitÂ

AGC Biologics offers deep industry expertise and uniquely customized services for the scale-up and cGMP manufacture of protein-based therapeutics (using mammalian and microbial systems), mRNA, pDNA, viral vector and cell therapy products, from pre-clinical to commercial production, including our proprietary CHEF1TM Expression System for efficient protein production with CHO cells. Our company DNA drives us to provide innovative solutions to partner with our customers in helping them reach their goals and accelerate their projects at our cGMP-compliant facilities in the US, Europe and Japan, as well as to facilitate approval and manage spending during the product lifecycle. We forge exceptionally strong partnerships with our customers and we never lose sight of our commitment to deliver reliable and compliant drug substance. Visit www.agcbio.com to learn more.

AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC's lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC's product pipeline includes six named ophthalmology development programs across five targets (X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration and blue cone monochromacy), two non-ophthalmology programs (alpha-1 antitrypsin deficiency and adrenoleukodystrophy) and AGTC is continuing to develop early research studies in additional indications. The company is also exploring genetic defects in cells in the inner ear that lead to deafness and expects to advance several product candidates into development within the next few years. AGTC employs a highly targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need, clinical feasibility and commercial potential. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products Find AGTC on YouTube at goo.gl/sMdJqX.

Ajinomoto is a global leader in the research, development, manufacture and sale of amino acid-based products for the pharmaceutical, nutraceutical, sports nutrition, health and beauty industries as well as food ingredients and consumer food products.

Akamis is a clinical-stage oncology company whose mission is to leverage its groundbreaking T-SIGn® platform to positively impact the lives of people living with cancer. We are developing a portfolio of solid tumor-targeted T-SIGn® therapeutics which aim to enable a patient’s own immune system to recognize, attack, and clear their cancer. Akamis’ growing pipeline of T-SIGn® therapeutics is anchored by its lead clinical-stage program, NG-350A, which is being investigated in an ongoing Phase 1 clinical study in patients with metastatic or advanced epithelial tumors. Akamis has a number of T-SIGn® platform-focused collaborations with leaders in the immuno-oncology field including BMS, Merck, and the Parker Institute for Cancer Immunotherapy (PICI)

Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss.

Alcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between FalconTM, the company’s proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering, and four novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone’s lead programs utilize X chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome, and gene replacement for the treatment of IGHMBP2-related disorders including spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot Marie Tooth disease type 2S (CMT2S).

Our mission is to transform the lives of people living with rare diseases and devastating conditions through the development and delivery of innovative medicines, as well as through supportive technologies and healthcare services. By continuing to deepen our understanding of rare disease, which began with our pioneering work in complement biology, we are able to innovate and evolve into new areas where there is great unmet need and opportunity to help patients and families fully live their best lives. Our culture is rooted in integrity, inclusiveness, and our dedication to joining and supporting the communities in which we live and work. We invest in and value people who believe in the importance of our purpose and understand what it takes to deliver on it. Alexion has over 3,000 talented colleagues dedicated to serving people living with rare diseases in more than 50 countries around the world. Our global headquarters are based in Boston, Massachusetts and our EMEA headquarters are in Zürich, Switzerland. We also have a Research Center of Excellence in New Haven, Connecticut, global supply chain and operations headquarters in Ireland, as well as local and regional operations in countries around the world. At Alexion, our passion drives us to continuously innovate and create meaningful value in all we do. In doing so, we change lives for the better – ours, people living with rare diseases, and the communities we serve. Every day.

Alia Therapeutics is a pioneering gene editing company with a vision to revolutionize medicine by curing genetic diseases at their roots. Alia’s versatile portfolio of novel CRISPR systems expands the range of targetable genomic sites and increases the precision of genetic modifications, bringing gene editing to the next level of specificity and safety, which are required for therapeutic applications.

Alpha Cognition is a clinical stage, biopharmaceutical company dedicated to developing novel treatments for under-served neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis (ALS). ALPHA-1062, a patented new chemical entity that has demonstrated safety and improved tolerability in human clinical trials. It is being developed as a new acetylcholine esterase inhibitor for the treatment of dementia of the Alzheimer’s type, with minimal gastrointestinal side-effects and novel routes of administration. ALPHA-0602 is a gene therapy program delivering progranulin, a neurotrophic protein. It is in preclinical development for the treatment of ALS. ALPHA-0602 is patented and has received Orphan Drug Designation from the FDA.

Teknova makes solutions possible. Since 1996, Teknova has been innovating the manufacture of critical reagents for the life sciences industry to accelerate the discovery and development of novel therapies that will help people live longer, healthier lives. We offer fully customizable solutions for every stage of the workflow, supporting industry leaders in cell and gene therapy, molecular diagnostics, and synthetic biology. Our fast turnaround of high-quality agar plates, microbial culture media, buffers and reagents, and water helps our customers scale seamlessly from RUO to GMP. Headquartered in Hollister, California, with over 200,000 square feet of state-of-the-art facilities, Teknova’s modular manufacturing platform was designed by our team of scientists, engineers, and quality control experts to efficiently produce the foundational ingredients for the discovery and commercialization of novel therapies.

Amarna Therapeutics is a privately held, pre-clinical biotech company developing transformative, potentially curative gene therapies for a range of both rare and prevalent diseases. The company is pioneering the development of gene therapies based on simian virus 40 (SV40), a macaque polyoma virus to which humans are immunologically naïve. Amarna has created a proprietary production cell line (SuperVero™) that, for the first time, makes it possible to produce SV40-derived vectors suitable for therapeutic use. Combining SuperVero™ with its genetically engineered SVec viral vector, the company’s fully-integrated gene therapy platform is truly unique in its intrinsic capability to deliver transgenes without eliciting immune responses to the vector nor to the transgene product. Leveraging this ‘natural’ non-immunogenicity feature of its gene delivery system in humans, Amarna is developing a broad pipeline of safe, effective and durable gene therapies focused on genetic disorders, autoimmune diseases and chronic inflammation. Founded in 2008, Amarna’s head office is based in Leiden Bio Science Park, the Netherlands, with a research subsidiary located in Seville, Spain.

Ambulero is a cell and gene therapy spin-out of the University of Miami focused on advancing new therapies to fight vascular disease. We are a platform company that can deliver the adhesion molecule E-selectin using different cell and synthetic vector technologies. We work on rare vascular diseases as well as more common indications effecting large patient populations.

Amicus Therapeutics, Inc., a biotechnology company, engages in the discovery, development, and commercialization of therapies to treat a range of rare and orphan diseases. The company offers Galafold, an orally administered small molecule pharmacological chaperone for the treatment of Fabry disease. It is also conducting Phase 3 (ATB200-03) clinical study of AT-GAA for Pompe disease. The company has collaboration and license agreements with Nationwide Children's Hospital and University of Pennsylvania. Amicus Therapeutics, Inc. was founded in 2002 and is headquartered in Philadelphia, Pennsylvania.

Amplo Biotechnology is a private Gene Therapy company focusing on therapy for diseases of the neuromuscular junction.

Andelyn Biosciences is a gene therapy CDMO dedicated to Turning Hope into Reality™. Our capabilities span viral vector process and analytical development, adherent and suspension based GMP drug substance manufacturing up to 2,000L, as well as GMP plasmid process development and GMP manufacturing. We are located in Columbus, OH, USA. With more than 20 years of pioneering experience in viral vector and vaccine manufacturing, Andelyn offers its clients direct access to globally recognized thought leaders with extensive expertise. We have produced GMP material for over 75 worldwide clinical trials in addition to over 450 cGMP clinical batches and more than 2,000 research-grade productions. Andelyn provides research and tox grade plasmids with an additional offering of GMP plasmid manufacturing, advanced quality systems, full regulatory support, semi-automated to automated fill stations, and supply chain vertical integration. Our 180,000 sq ft clinical and commercial manufacturing facility comprises 16 upstream production suites for customization of new and tech transferred programs. Andelyn offers clinical through commercial-scale capabilities that will help accelerate the development and manufacturing of innovative therapies to bring more treatments to more patients.

At Anlong Bio, patients are our priority. Since launching, our goal has been to deliver transformative gene therapy medicines to patients faster and at low cost, in China and globally. We aim to provide unmatched access to treatments for patients through our extensive resources, our experienced leadership and advisory teams, and our trusted network of partners.

Antibodies Incorporated is a biotechnology company that specializes in manufacturing and distributing monoclonal and polyclonal antibodies, diagnostic reagents, diagnostic kits, and developing ImmunoAssays.

Apertura is a biotechnology company opening opportunities in gene therapy for treating debilitating diseases with limited options. We are pioneering next-generation AAV gene therapies by simultaneously engineering capsids, regulatory elements, and payloads to overcome current limitations in genetic medicine. Founded on technologies from the Broad Institute and Harvard University, and with support from Deerfield Management Company, we are located at the Cure in New York City.

Applied Viromics is a biotech company aiming to support early drug discovery and development activities. Applied Viromics specializes in custom-made recombinant viral vectors for efficient gene delivery both in vitro and in vivo.

Arbor Bio is a next-generation gene editing company focused on discovering and developing potentially curative genomic medicines, with the most extensive toolbox of proprietary genomic editors in the industry. We have the unique ability to work backward from disease pathology and choose the optimal editors or combination of editors for the disease in question.

ArcticZymes Technologies ASA (OSE: AZT) is a global leader in supplying best-in-class enzyme technologies to commercial partners who develop therapeutics, in vitro diagnostics, and molecular research kit products. Our mission is to make our customers lives easier by unlocking new solutions to enhance quality of life and create a more sustainable healthy world. We provide our customers with cold-adapted enzymes from the arctic environment with unique features including heat lability and activity in challenging environments, as well as, customised engineered novel features which can be easily integrated to fuel their innovations. For more information, please visit www.arcticzymes.com

ArcticZymes Technologies ASA (OSE: AZT) is a global leader in supplying best-in-class enzyme technologies to commercial partners who develop therapeutics, in vitro diagnostics, and molecular research kit products. Our mission is to make our customers lives easier by unlocking new solutions to enhance quality of life and create a more sustainable healthy world. We provide our customers with cold-adapted enzymes from the arctic environment with unique features including heat lability and activity in challenging environments, as well as, customised engineered novel features which can be easily integrated to fuel their innovations. For more information, please visit www.arcticzymes.com

Armatus Bio is a biotechnology company that specializes in precision gene therapy for raising expectations in FSHD.

Ascend is a development partner that supports life-science innovators in bringing gene therapy products to patients. Our team has deep expertise in gene therapy development and commercialization to offer unparalleled collaboration while working to develop your products from the bench through the clinic and beyond. Scientific innovation driven by quality and responsibility are at the core of our business. To set a new standard in the industry, we have invested in a world-class analytical toolkit within our flexible and scalable AAV platform of next-gen technologies, all built to find the perfect balance of yield and quality for each process. Let’s aim higher and develop the next wave of accessible life-saving therapies together!

The company supports cell and gene therapy manufacturing and process development projects from design through to clinical and commercial scale.

ASC Therapeutics focuses on the development of curative therapies for rare diseases. Our gene, CRISPR and cell therapies were developed at Applied StemCell, a life sciences company for over 12 years, including proprietary gene editing technologies. Our development pipeline includes several preclinical stage projects focusing on monogenic blood disorders that exhibit a high genetic penetrance and conditions requiring immunomodulatory interventions. Contact us for partnership opportunities

Asgard Therapeutics is a private biotech company exploring the application of direct cell reprogramming technologies for cancer immunotherapies. Formed as a spin-off from Lund University, the Company is pioneering a gene therapy approach based on its proprietary TrojanDC technology, designed to set in motion immune responses based on the biological properties of professional antigen presenting cells. Backed by Novo Holdings, Boehringer Ingelheim Venture Fund and Industrifonden, Asgard Therapeutics aims to build a pipeline of personalized cancer immunotherapies optimized to each unique patient.

Asimov's mission is to advance humanity's ability to design living systems, enabling biotechnologies with outsized societal benefit. The company is developing a synthetic biology platform from cells to software to design and manufacture next-generation therapeutics, including cell & gene therapies, through a combination of products, services, & collaborations. Founded by bioengineers from MIT and Boston University and headquartered in Boston, the company has raised over $200 million.

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for congestive heart failure, Huntington’s disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. Learn more at askbio.com. Vision - Pioneering science to create transformative molecular medicines. Mission - Lead innovative science and drive clinical outcomes to transform people's lives. Values: • Be a Pioneer. We are not afraid of the impossible and to innovate to make gene therapies accessible to those in need. • Cultivate Collaboration. Strive to be the best teammate, actively listen, openly communicate, and embrace diverse points of view. • Embrace Responsibility. We are humbled by the enormity of our mission. We hold a relentless commitment to advance science and clinical outcomes for our patients, families, and caregivers. • Raise the Bar. Continuously drive improvements and efficiencies. Seek and provide constructive feedback. Have a bias for learning and action. • Act with Uncompromising Integrity. Be honest, transparent, and committed to doing what’s right in every situation. Make clear commitments and follow through.

Aspa Therapeutics, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on developing a treatment for Canavan disease, a devastating inherited condition without an FDA-approved therapy. Aspa is led by a team of veteran biotechnology executives. Together with patients and physicians, the company aims to bring a safe and effective treatment for Canavan disease to patients as quickly as possible.

Astellas Pharma US, Inc. is an affiliate of Tokyo-based Astellas Pharma Inc., a top 20 global pharmaceutical research company. Astellas is committed to turning innovative science into medical solutions that bring value and hope to patients and their families. Keeping our focus on addressing unmet medical needs and conducting our business with ethics and integrity enables us to improve the health of people throughout the Americas and around the world. All content developed by Astellas Pharma US, Inc. and intended for U.S. audiences.

Driven by innovative science and our entrepreneurial culture, we are focused on the delivery of life-changing medicines that are fuelling growth and contributing value to patients and society.

Atamyo Therapeutics is focused on the development of a new generation of safe and effective gene therapies for neuromuscular diseases. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD).

Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which leverages the company’s novel spreading capsid AAV.SPR, is being evaluated in XLRS patients in a Phase I/II clinical trial known as the LIGHTHOUSE study. The company’s additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with LCA1 have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 12 months post-treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise.

Avantea is a laboratory of advanced technologies for biotechnology research and animal reproduction. The activity of this laboratory began in 1991 with Cesare Galli and Giovanna Lazzari. In 2008 it became Avantea Srl, inheriting all the skills and expertise built in about twenty years of activity and the know-how developed through years of research conducted in the biomedical and animal reproduction fields. Credibility and experience make Avantea a reliable and highly specialized center, which aims to find innovative solutions in the field of reproductive biotechnology for livestock breeding and biomedicine. Avantea research activity is assessed by an independent Ethics Committee, which includes leading experts in animal health and welfare, bioethics, genetics, law and forensic medicine and it is in agreement with the guidelines for the application of Article 48 of the Ethics Code of the National Federation of Italian Veterinarians. Avantea has a Quality Management System and obtained the Quality Certification according to UNI EN 9001:2008.

Aves Labs produces high-affinity, custom chicken IgY and other immunoreagents for biomedical research and antibody manufacturing.

At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and our suite of proprietary gene therapy platforms and delivery technologies, we are working relentlessly to overcome the challenges of delivering the right drug to the right place. Our innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute.

Axovia Therapeutics is dedicated to the research and development of novel AAV gene therapies that target key aspects of ciliopathies, addressing the devastating impact of the disease head-on for patients, their families and caregivers. Axovia is currently developing therapies for patients with mutations in the BBS1 gene which manifests as Bardet Biedl Syndrome. Our initial goal is to halt the progression of BBS by targeting the most debilitating aspects of the neurological, metabolic and retinal dysfunctions.

Batavia Biosciences is a science-based CDMO that offers personalized services that fully support all the stages of biopharmaceutical development, from cloning to GMP manufacture and release of clinical material for phase I/II studies. We are not your standard CDMO - our customers regularly refer to us as their product development partner and thought partner. We focus on the early stages of product development with services ranging from DNA cloning, mammalian cell line generation, upstream process development, purification development, product characterization to clinical manufacturing. Headquartered in Leiden, The Netherlands, Batavia Biosciences is privileged to have strong strategic partners in both the EU and US. Batavia Biosciences aims to significantly contribute to ease human suffering from infectious disease and cancer.

Bayer is a global enterprise with core competencies in the Life Science fields of health care and agriculture. Our products and services are designed to benefit people and improve their quality of life. At the same time, we aim to create value through innovation, growth and high earning power. Our products help address some of today’s biggest challenges, including global population growth, an aging society and the need to make efficient – and, wherever possible, sustainable – use of natural resources. In line with our mission “Bayer: Science For A Better Life,” we aim to improve people’s quality of life by preventing, alleviating or curing diseases. We also help provide an adequate supply of high-quality food, feed and renewable plant-based raw materials. For these endeavors, we focus on developing and successfully commercializing innovative products and solutions based on scientific knowledge.

Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to restore and improve the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another in-licensed from the University of Oxford targeting cone-rod dystrophy (CRD), an inherited retinal disease. Lead development candidate AGTC-501, is a gene therapy program in Phase II clinical trials for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises.

Beam Therapeutics Inc., a biotechnology company, engages in developing precision genetic medicines for patients suffering from serious diseases in the United States. The company is developing therapies for the development of sickle cell disease and beta-thalassemia; CAR-T cell therapies for pediatric T-cell acute lymphoblastic leukemia and pediatric acute myeloid leukemia; therapies for alpha-1 antitrypsin deficiency and glycogen storage disorder 1A; and therapies for ocular and central nervous system disorders. It has a research and clinical collaboration agreement with Magenta Therapeutics, Inc. Beam Therapeutics Inc. was founded in 2017 and is based in Cambridge, Massachusetts.

Be Bio is pioneering Engineered B Cell Medicines (BCMs) to dramatically improve the lives of patients who are living with Hemophilia B and other genetic diseases, cancer, and other serious conditions. With eyes locked on the patient, our team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborate to create a bold new class of cell therapies.

Bedrock Therapeutics is an AAV gene therapy research and development company developing platform and disease specific technologies for the treatment of ocular, hematologic, and other diseases. Drs. Matt Hirsch, Chenwen Li, Bryan Sutton, and Brian Gilger are co-founders of Bedrock. Drs. Gilger and Hirsch oversee the de novo design and validation of ocular assets, and Drs. Li and Sutton oversee basics aspects of the various host immune responses to AAV vectors and protein engineering and design.

Founded in 2018, Belief BioMed is aiming to become a globally leading gene therapy company by being committed to providing innovative therapies with improved efficacy for monogenic disorder diseases, age-related degenerative diseases, and certain malignant diseases through its AAV vector technology from early discovery to commercialization. The R&D and production strengths of Belief BioMed have been recognized by top investment institutions and enterprises. Belief BioMed has offices, research centers and manufacturing facilities in Shanghai, Hong Kong, Beijing and Suzhou China and North Carolina US.

Benitec is a biotechnology company developing a proprietary therapeutic technology platform that combines RNA interference (RNAi) with gene therapy with a goal of providing sustained, long-lasting silencing of disease-causing genes from a single administration. Benitec is using its technology, called DNA-directed RNA interference (ddRNAi) to develop a pipeline of product candidates for the treatment of several chronic and life-threatening human diseases , including oculopharyngeal muscular dystrophy (OPMD) and hepatitis B. By combining the specificity and gene silencing effect of RNAi with gene therapy, ddRNAi has the potential to produce long-lasting silencing of disease-causing genes from a single administration, which could minimize the requirement for patients to take regular doses of medicine. Benitec’s objective is to become the leader in discovering, developing, clinically validating and commercializing ddRNAi-based therapeutics for a range of human diseases with high unmet clinical need or large patient populations and, as a result, provide a better life for patients with these diseases.

Bielefeld University is a Higher Education institution that provides academic programs and conducts research.

BioAgilytix is a biotechnology company that specializes in bioanalysis for the biopharmaceutical and life sciences industries.

Through cutting-edge science and medicine, Biogen discovers, develops and delivers innovative therapies worldwide for people living with serious neurological and neurodegenerative diseases. Founded in 1978, Biogen is a pioneer in biotechnology and today the Company has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first and only approved treatment for spinal muscular atrophy, and is at the forefront of neurology research for conditions including Alzheimer’s disease, Parkinson’s disease and amyotrophic lateral sclerosis. Biogen also manufactures and commercializes biosimilars of advanced biologics. With approximately 7000 people worldwide, we are truly a global organization, headquartered in Cambridge, Massachusetts, which is also home to our research operations. Our international headquarters are based in Zug, Switzerland and we have world-class manufacturing facilities in North Carolina and Denmark. We offer therapies globally through direct affiliate presence in 30 countries and a network of distribution partners in over 50 additional countries.

BioMap is an AI for life sciences company, unmasking nature’s rules to generate proteins for a wide range of therapeutic and industrial applications. We build super-scale Foundation models supported by access to unrivaled computational power through our strategic collaboration with Baidu, with whom we share our Founder and Chairman, Robin Li. We supplement our AI capabilities with a leading team of scientists, disease experts and cutting-edge high-throughput labs which generate large volumes of high quality proprietary data for our AI models. We partner with pharma, biotechs and industry to enable their discovery ambitions across a wide range of therapeutic and synthetic biology modalities through access to our AI technology platform, with a variety of flexible collaboration models. Our mission is to leverage AI to radically improve lives.

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.

Contract Manufacturing & Analytical

Biomiga, Inc. is an innovative biotech research and service company based in San Diego, California. Our mission is to provide the highest quality laboratory products while maintaining the best possible service and support to our end-users. Our products maximize efficiency and cost-effectiveness through our cutting-edge technology and process. Biomiga owns the manufacturing process, enabling us to control both the quality of the products as well as the price. Our products are more reliable and effective while often costing less than half the price of our competitors. Biomiga can customize its products to meet a client’s exact specifications and requirements. Biomiga provides full service support to all our clients through the entire process. Our support and sales staffs are available for consultation 24 hours a day, 7 days a week. We stand behind all our products with a full guarantee. Our mission is to ensure our client’s satisfaction. Biomiga emphasizes on product development. Our R&D team is highly trained in the biological sciences to quickly develop new products. Our products have been proven to meet our clients' high demands with greater cost savings and effectiveness. EzgeneTM and ViraTrapTM are just some examples.

BioNTech is a global next generation immunotherapy company aspiring to translate science into survival. Around 6.100 pioneers currently work with us, employing a wide array of computational discovery and therapeutic drug platforms for the rapid development of novel biopharmaceuticals. Our broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor (CAR) T cells, several protein-based therapeutics, including bispecific immune checkpoint modulators, targeted cancer antibodies and antibody-drug conjugate (ADC) therapeutics, as well as small molecules.   Based on our deep expertise in mRNA vaccine development and in-house manufacturing capabilities, we are developing multiple mRNA vaccine candidates for a range of infectious diseases alongside its diverse oncology pipeline. To learn more about us, please visit www.BioNTech.com. Our privacy statement can be found here:  https://www.biontech.com/int/en/home/data-privacy-statement.html Our imprint can be found: https://www.biontech.com/int/en/home/imprint.html

BIOQUAL, Inc. is fully accredited by the Association for the Assessment and Accreditation of Laboratory Animal Care International (AAALAC) and has a long history of providing state-of-the-art facilities and trained, experienced research scientists, veterinarians and animal care personnel for clients involved in infectious disease investigations, cancer research, and a wide variety of other in vivo and in vitro studies.

Research & Diagnostic Products. Bio-Rad is partnering with Biodesix on a test for SARS-CoV-2.EUA (05/2020) for a digital PCR molecular diagnostic test for SARS-CoV-2, the virus that causes coronavirus disease COVID-19. The 2019-nCoV CDC ddPCR Triplex Probe Assay is now authorized for use on the firm's QX200 and QXDx Droplet Digital PCR systems.

BioViva USA is dedicated to improving healthy human longevity through bioinformatics. The Biovault bioinformatics platform is contributing to health predictions and recommendations, precision medicine, and the discovery of novel biomarkers by applying state of the art computational methods on vastcollections of biological data.BioViva is partnered with Integrated Health Systems (IHS). IHS is an innovative company specializing in cell and gene therapy, aprivate care company supporting health professionals working with groundbreaking treatments and therapeutics to support healthy longevity. Theywork exclusively with regenerative medicine specialists targeting the aging process.BioViva is committed to:Bridging stakeholders with biotechnologyFacilitating the development of treatments and therapeutics through the BioViva platform, designed to be readily browsable by longevity researchers.Accelerating the path to human trials for life-saving gene therapies.Providing biotechnology companies, medical researchers, and patients access to one another, expediting research and development.The Biovault will continuously update user profiles, including changes in relevant biomarkers. BioViva is utilizing the latest machine learning algorithms to formulate hypotheses and validate findings, thereby paving the way for optimal combinatorial regimens addressing the root causes of the aging process.

Bloomsbury Genetic Therapies ("Bloomsbury") is a clinical-stage biotechnology company, developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies. The Company was spun out of University College London and launched in October 2022 with funding from UCL Technology Fund. Bloomsbury is building a pipeline of highly differentiated first- or best-in-class programs.

Borea Therapeutics develops next-generation gene therapy vectors using a knowledge-based capsid design platform

BIB is a full service CDMO partner offering cGMP and non-GMP mammalian, microbial, gene therapy and cell therapy services.

BrainEver is a biopharmaceutical company developing a pipeline of novel therapies for patients with age-related neurodegenerative diseases, such as Charcot disease, Parkinson’s, and glaucoma, the second leading cause of blindness. Our mission is to develop recombinant human Homeoproteins (HPs) to halt disease progression by stopping neuronal loss and durably restoring the physiology and metabolism of the remaining neurons. BrainEver was co-founded by Bernard Gilly (iBionext) and Alain Prochiantz (Collège de France). Our innovative therapeutic approach stems from the pioneering work of Alain Prochiantz and his team on brain development and physiology. Our lead project, BREN02, focuses on developing recombinant human Engrailed-1 (hEN1) for the treatment of patients with Amyotrophic Lateral Sclerosis (ALS). Building on the highly promising results of our preclinical studies, we are preparing to launch clinical trials by 2025. To fund this next phase and secure market approval by 2027, we are opening our capital to those eager to join a unique human and scientific journey. We are supported by prominent investors such as Bpifrance, Ibionext, and Turenne Capital. Now, we aim to raise €35M, with €5M available for participation through the crowdfunding platform Tudigo. Become a BrainEver shareholder 👉 https://tudigo.typeform.com/brainever

Established in 2012, BravoVax is located at BravoVax Building, 858 Gaoxin Avenue in the state-level biopharma development zone, Donghu hi-tech park of Wuhan, China. Relying on the experienced management team and international channels from parent company BravoBio and strong support from Humanwell Healthcare Group, one of the top 20 Chinese pharmaceuticals, we have kept the momentum in its development, maintaining fast growth. We are committed to the research and development of human vaccines and other biological products. The new pilot facility and R&D center are built at Wuhan biopharaceutical industrial park as per cGMP. All staffs hold a college and above degree, and more than 30% of them have master and above degrees as well as senior professional titles.

BridgeBio Pharma, Inc. engages in the discovery, development, and delivery of various medicines for genetic diseases. The company has a pipeline of 30 development programs that include product candidates ranging from early discovery to late-stage development. Its products in development programs include BBP-265, a small molecule stabilizer of transthyretin, or TTR, that is in an ongoing Phase 3 clinical trial for the treatment of TTR amyloidosis-cardiomyopathy, or ATTR-CM; BBP-831, a small molecule selective FGFR1-3 inhibitor which is an ongoing Phase 2 clinical trial for the treatment of achondroplasia in pediatric patients; an AAV5 gene transfer product candidate for the treatment of congenital adrenal hyperplasia, or CAH, driven by 21-hydroxylase deficiency, or 21OHD; and Encaleret, a small molecule antagonist of the calcium sensing receptor, or CaSR, an ongoing phase 2 proof-of-concept clinical trial for Autosomal Dominant Hypocalcemia Type 1, or ADH1. The company also engages in developing products for Mendelian, genetic dermatology, oncology, and gene therapy diseases. BridgeBio Pharma, Inc. has license and collaboration agreements with the Leland Stanford Junior University; The Regents of the University of California; Leidos Biomedical Research, Inc.; Johns Hopkins University and University of Florida; University of Colorado Anschutz Medical Campus; Salk Institute for Biological Studies; Maze Therapeutics; and UC San Francisco. The company was founded in 2015 and is headquartered in Palo Alto, California.

Bristol-Myers Squibb Company discovers, develops, licenses, manufactures, and markets biopharmaceutical products worldwide. It offers products for hematology, oncology, cardiovascular, immunology, fibrotic, neuroscience, and covid-19 diseases. The company’s products include Revlimid, an oral immunomodulatory drug for the treatment of multiple myeloma; Eliquis, an oral inhibitor for reduction in risk of stroke/systemic embolism in NVAF, and for the treatment of DVT/PE; Opdivo for anti-cancer indications; Pomalyst/Imnovid indicated for patients with multiple myeloma; and Orencia for adult patients with active RA and psoriatic arthritis. It also provides Sprycel for the treatment of Philadelphia chromosome-positive chronic myeloid leukemia; Yervoy for the treatment of patients with unresectable or metastatic melanoma; Abraxane, a protein-bound chemotherapy product; Reblozyl for the treatment of anemia in adult patients with beta thalassemia; and Empliciti for the treatment of multiple myeloma. In addition, the company offers Zeposia to treat relapsing forms of multiple sclerosis; Breyanzi, a CD19-directed genetically modified autologous T cell immunotherapy for the treatment of adult patients with relapsed or refractory large B-cell lymphoma; Inrebic, an oral kinase inhibitor indicated for the treatment of adult patients with myelofibrosis; and Onureg for the treatment of adult patients with AML. It sells products to wholesalers, distributors, pharmacies, retailers, hospitals, clinics, and government agencies. The company was formerly known as Bristol-Myers Company. The company was founded in 1887 and is headquartered in New York, New York.

At BRL Medicine Inc., we aim to become the world's leading Cellular & Gene pharmaceutical company in the era of new commercial civilization, with the mission of "Through innovation led by gene editing, develop breakthrough human therapeutics and benefit the whole world". With its independent R&D center and the "Shanghai Research Center for Gene Editing and Cell Therapy", jointly established by BRL Medicine and university, BRL Medicine has obtained more than 100 patent achievements, and initiated 5 Investigator-Initiated clinical Trials (IIT) in 8 leading hospitals in China, with many pipeline projects have entered into IND application stage. Among them, gene editing projects for β-thalassemia, PD1 knockout non-viral targeted integration CAR-T and UCART have achieved excellent clinical results and have published several academic papers in famous academic journals such as Nature Medicine and Nature biotechnology. BRL Medicine has built five technology platforms with independent intellectual property rights, including The Gene Editing Technology Innovation Platform, Hematopoietic Stem Cell Platform, The Quikin CAR-T Platform, Universal Cell Platform and Enhanced T Cell Platforms, and has a 7000 square meter GMP pilot plant and an operating team of more than 200 people, which strongly guarantees the rapid transformation and application of innovative research. BRL Medicine continuously promotes rapid update and iteration of R&D products through patient needs and clinical feedback. Driven by the era of new commercial civilization, BRL Medicine holds an open, shared, and win-win attitude, and works with global innovative biomedical ecological chain companies to accelerate the transformation and implementation of innovative drugs, so as to benefit global patients with genetic diseases and malignant tumors!

Broad Institute of MIT and Harvard was launched in 2004 to improve human health by using genomics to advance our understanding of the biology and treatment of human disease, and to help lay the groundwork for a new generation of therapies.COVID-19: EUA (03/2021) for CRSP SARS-CoV-2 Real-time Reverse Transcriptase-PCR Diagnostic Assay

Calidi Biotherapeutics is a clinical-stage biopharmaceutical company revolutionizing oncolytic viral therapies with stem cell-based platforms.

CANbridge Pharmaceuticals is a pharmaceutical company that focuses on developing and commercializing innovative medicines to address unmet medical needs.

At Candel Therapeutics, we are developing immunotherapies that induce immunogenic cell death in cancer cells at the site of injection, unmasking tumor neo-antigens within an activated microenvironment. This process leads to a systemic, durable immune response against the tumor, with the potential to change disease outcomes across a variety of indications. Candel’s products are designed to improve survival while maintaining quality of life – from early- to late-stage disease.

CanSino Biologics Inc. (CanSinoBIO, SHSE: 688185, HKEX:06185) is an innovative biopharmaceutical company dedicated to exploring best solutions to the prevention of diseases through cutting-edge research & development, advanced manufacturing and commercialization of innovative vaccine products for human use worldwide. Since its establishment in Tianjin, China in 2009, CanSinoBIO has experienced tremendous growth with 17 vaccines preventing 12 diseases, including approved vaccines for Ebola virus disease (Ad5-EBOV), COVID-19 (Ad5-nCoV, trade name: Convidecia), and meningitis (MCV2, trade name: Menphecia). CanSinoBIO has been listed on the Main Board of Hong Kong Exchange and Clearing Limited (HKEx) in March 2019. A year later, CanSinoBIO has successfully listed on the Sci-Tech Innovation Board (STAR Market) of the Shanghai Stock Exchange, making it the first "A+H" dual listing vaccine company. CanSinoBIO is focusing on continually expanding manufacturing capacity for its current vaccine candidates and further enhancing the competitiveness and the scope of its portfolio by promoting the R&D of new vaccine candidates. Leveraging broad experiences of our accomplished team of scientists and business leaders who had previously held technical and senior management positions at many of the leading pharmaceutical companies in the world, including Sanofi Pasteur, Astra Zeneca, Wyeth (now Pfizer) and CNBG (China), CanSinoBIO has developed five key platform technologies, including viral vector-based technology, synthetic vaccine technology, protein structure design and recombinant technology, mRNA technology as well as formulation and delivery technology.

CAPACITÉS is the subsidiary of University of Nantes, dedicated to the development of research. Specialized in engineering of innovative projects, CAPACITÉS exploits the latest scientific discoveries necessary for the performance of its customers. Springboard to differentiate in the ever-changing markets, areas of activity extend to technological and engineering sciences, human and social sciences and health. The activities of CAPACITÉS have advanced expertise on specific and cross-cutting themes. From idea generation to their implementation, CAPACITÉS impulse with its customers a strong value creation process through the development of R&D of high level dedicated to nine economic sectors.

Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders. Capsida's technology allows for the targeted penetration of cells and organs, while limiting collateral impact on non-targeted cells and organs, especially the liver. This technology allows for the delivery of the gene therapy in a non-invasive way through intravenous administration. Capsida's technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of life-threatening genetic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida's technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development experience. Capsida's biologically driven, high-throughput AAV engineering and cargo optimization platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology.

Capsigen is a leader in the engineering of AAV capsids for use in gene therapies, opening the path to permanent cures for patients suffering from intractable illness. Capsigen is enabling a new era of genetic medicines based on a disease-first approach. Powered by our TRADE™ and data science platforms, we engineer vectors with the highest possible potency and cell-type specificity. Developing genetic medicines that are tailored to the unique biology involved in the disease ensures that the therapy can bring the maximum benefit to patients. Our team is driven in its pursuit to deliver on this promise to the millions of people depending on the advancement of science to discover new cures.

Caribou Biosciences, Inc., a clinical-stage biopharmaceutical company, engages in the development of genome-edited allogeneic cell therapies for the treatment of hematologic malignancies and solid tumors in the United States and internationally. Its lead product candidates are CB-010, an allogeneic anti-CD19 CAR-T cell therapy that is in phase 1 clinical trial to treat relapsed or refractory B cell non-Hodgkin lymphoma; and CB-011, an allogeneic anti-BCMA CAR-T cell therapy for the treatment of relapsed or refractory multiple myeloma. The company also develops CB-012, an allogeneic anti-CD371 CAR-T cell therapy for the treatment of relapsed or refractory acute myeloid leukemia; and CB-020, an allogeneic CAR-NK cell therapy for the treatment of solid tumors. Caribou Biosciences has collaboration with AbbVie Manufacturing Management Unlimited Company to develop CAR-T cell therapies. The company was incorporated in 2011 and is headquartered in Berkeley, California.

Carnegie Mellon University is a Higher Education institution that focuses on research and technology innovation.

Cartesian Therapeutics is a fully integrated, clinical-stage biopharmaceutical company developing novel cell and gene immunotherapies to treat cancer and autoimmune diseases.

The Offshore Renewable Energy (ORE) Catapult forms part of an elite network of eight technology and innovation centres established and overseen by Innovate UK, which represents a £1bn public and private sector investment over the next five years. ORE Catapult is the UK's flagship technology innovation and research centre for offshore wind, wave and tidal energy. We deliver prioritised research underpinned by world-class test and demonstration facilities, collaborating with industry, academia and Government to reduce the cost of offshore renewable energy and create UK economic benefit.

Cellastra Inc., a developer of disruptive gene therapies, is a private biotech company based in the San Francisco Financial District. It was founded and managed by proven industry executives with a track record of execution. Cellastra’s mission is to develop Disruptive, First or Best in Class Gene Therapies. Cellastra Proprietary Technology - Gene Vector- Anti-scarring peptide: Cellastra is developing viral gene vector-mediated intervention to address an area of great unmet medical needs in scarless wound healing after surgery and respiratory infections: 1. Surgery: Prevent excessive dermal scarring, e.g., after breast augmentation surgery, and C section. 2. Respiratory Infections: Prevent an excessive immune response, tissue damage, and scarring/fibrosis in high-risk patient groups with respiratory infections such as COVID-19 (coronavirus disease 2019) and RSV (respiratory syncytial virus). Scarless wound healing can be achieved by using a gene vector encoding for endogenous continuous production of the anti-scarring peptides at the site of injury. Thus, two gene vectors are planned for development (Cellexa applied under the skin after surgery and Virexa inhaled into the lungs in COVID-19 and other respiratory infections). The development of these vectors is built and expands upon a foundation of scientific proof of concept data of the anti-scarring peptide ensereptide.

Cellectis is a global clinical-stage biopharmaceutical company. Pioneers and innovators in our field, our mission is to develop innovative treatments for patients with unmet medical needs. With 25 years of expertise, we have the best-in-class gene editing platform focusing on immuno-oncology, and gene therapy. Through our efficient and precise TALEN® technology, we create allogeneic CAR-T cells capable of recognizing and combating cancer cells. Today, our three clinical programs target patients with B-cell acute lymphoblastic leukemia (B-ALL), non-Hodgkin lymphoma (NHL) and acute myeloid leukemia (AML). We are fully integrated and a leader in end-to-end gene editing, allogeneic CAR T-cell companies. With our in-house manufacturing, we control our gene and cell therapy process from start to finish with starting materials produced in Paris (France) and CAR-T therapy products created in Raleigh, NC (USA). We also have several ongoing strong collaborations, based on our TALEN® technology, with leading cell & gene therapy companies, including our recent partnership with AstraZeneca, to develop new product candidates in oncology, immunology, and treatment of rare diseases. At Cellectis, we are committed to a cure. Cellectis headquarters are in Paris, France, with additional locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more about us, visit our website: www.cellectis.com. To learn more about our community guidelines, visit: http://www.cellectis.com/en/social-media-guidelines/ Follow our other social media accounts: @cellectis on LinkedIn on X (formerly Twitter) . TALEN® is a registered trademark owned by the Cellectis Group.

Center for Breakthrough Medicines is an innovative cell and gene therapy organization focused on contract development and manufacturing organization located in the heart of “Cellicon Valley.”

Century Therapeutics, Inc., a biotechnology company, develops transformative allogeneic cell therapies for the treatment of solid tumor and hematological malignancies. The company’s lead product candidate is CNTY-101, an allogeneic, induced pluripotent stem cells (iPSCs)-derived chimeric antigen receptors (CAR)-iNK cell therapy targeting CD19 for relapsed, refractory B-cell lymphoma. It is also developing CNTY-103, a CAR-iNK candidate targeting CD133 + EGFR for recurrent glioblastoma; CNTY-102, a CAR-iT or CAR-iNK candidate targeting CD19 + CD79b for relapsed, refractory B-cell lymphoma and other B-cell malignancies; and CNTY-104, a CAR-iT or CAR-iNK multi-specific candidate for acute myeloid leukemia. Century Therapeutics, Inc. has a research collaboration with Outpace Bio, Inc. for CAR engineering. The company was founded in 2018 and is headquartered in Philadelphia, Pennsylvania.

CG Oncology, Inc. (Nasdaq: CGON), is a late-stage clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients afflicted with bladder cancer. We see a world where urologic cancer patients can benefit from our innovative oncolytic immunotherapies to live with dignity and have an enhanced quality of life. Our lead candidate, cretostimogene grenadenorepvec, is an intravesically delivered oncolytic immunotherapy agent in a Phase 3 trial for the treatment of BCG-unresponsive non-muscle invasive bladder cancer. Cretostimogene grenadenorepvec is also in a Phase 2 study in combination with KEYTRUDA® (pembrolizumab) in the same indication. Other types of bladder cancer are being evaluated with cretostimogene grenadenorepvec in combination with OPDIVO® (nivolumab).

At Charles River, we are passionate about our role in improving the quality of people’s lives. Our mission, our excellent science and our strong sense of purpose guides us in all that we do, and we approach each day with the knowledge that our work helps to improve the health and well-being of many across the globe.

Children's Mercy Hospital is a 367-bed comprehensive pediatric medical center in Kansas City, Missouri that integrates clinical care, research and medical education to provide care for patients ages birth to 21.

Chimerna Therapeutics is a biotech company focused on RNA therapeutics. Their platform for generating circular RNA overcomes the major limitations of RNA therapeutics, such as stability and abundance.

CHO Plus is creating regulatory compliant genetically engineered mammalian cells to be used by biopharmaceutical companies for therapeutic protein manufacturing. Early proof of concept experiments demonstrate CHO Plus cells to be 4.5-fold more productive than parent cells in model system. The CHO Plus approach includes patented methods for genetically engineering mammalian cells; proprietary and patented targeted transfection technology; and efficient clone screening technology.

Circio Holding ASA is a clinical stage company developing immune activators to target difficult-to-treat diseases. The company’s mission is to deploy vector-based technology to create a novel class of targeted, multi-functional circRNA medicines. It has established a cutting-edge circular RNA (circRNA) program and is collaborating with Prof Michael Uhlin and a team at the Karolinska Institutet in Stockholm with the intent to develop next generation RNA therapeutics for various applications. The company also has a mutant RAS cancer vaccine program, with two collaborative clinical trials underway in the USA and Norway. It is currently looking at options for the financing of a phase 2 study for ONCOS-102, a genetically modified oncolytic adenovirus.

Cirsium Biosciences is democratizing gene therapy through transformative viral vector production methods and systems.

CLAIRIgene is a preclinical stage biopharmaceutical company focused on development gene therapies for neurodegenerative diseases. Founded by innovative scientists with decades of experience, CLAIRIgene is committed to rapidly advancing their therapeutics to the clinic.

Clearside Biomedical, Inc., a biopharmaceutical company, engages in the developing and delivering treatments that restore and preserve vision for people with serious eye diseases. Its product includes CLS-AX, an axitinib for suprachoroidal injection which is in Phase 1/2a clinical trial. It also develops XIPERE, a triamcinolone acetonide suprachoroidal injectable suspension for the treatment of macular edema associated with uveitis, diabetic macular edema, and macular edema associated with retinal vein occlusion. The company was incorporated in 2011 and is headquartered in Alpharetta, Georgia.

CLS Therapeutics is a biopharmaceutical firm that provides transformative gene therapy solutions for cancer patients.

Coastar Therapeutics is a pre-clinical biotech company developing biological payload delivery technologies for cancer immunotherapy and gene therapies. Our proprietary ENHEnS technology can be used to coat biological payloads with cell membranes, which help them evade recognition and clearance by the immune system and be successfully delivered to tumor or other disease sites.

Coave Therapeutics is a clinical stage biotechnology company focused on developing life changing gene therapies in rare Ocular and CNS (Central Nervous System) diseases. We are pushing the boundaries of gene therapy through application of our AAV-Ligand Conjugates platform (ALIGATER) that enables targeted delivery, enhanced gene transduction and tissue distribution to improve the effectiveness of advanced gene therapies for rare diseases. Our strategy is to advance a pipeline of novel therapies targeting rare ocular and CNS diseases where targeted gene therapy has the potential to be most effective, where there is a clear unmet need and where success will provide rapid validation of our ALIGATER platform.

Cognigenics develops innovative RNA-based gene therapies targeting memory loss, cognitive decline, anxiety associated with mild cognitive impairment and other neurocognitive and neuropsychiatric disorders. Our intranasal delivery platform targets neuronal receptors in the brain involved in CNS disorders including anxiety, and Mild Cognitive Impairment.

Complement Therapeutics GmbH (CTx) is an early-stage biotechnology company focused on the research and development of novel therapeutics for complement-mediated diseases. The Company is a spinout from the University of Manchester and is based on the pioneering research of its founders into novel targets within the complement cascade. Our lead investigational product (CTx001) is being evaluated as a potential gene therapy for dry age-related macular degeneration/geographic atrophy, a leading cause of blindness. Additional programmes will evaluate potential therapeutic opportunities in other complement-mediated conditions. The Company is also developing a unique quantification methodology, the Complement Precision Medicine (CPM) platform, to enable quantification of over 30 complement cascade proteins enabling more precise diagnosis and monitoring of disease.

Concinnity is developing an AI-driven RNA design platform to build gene control systems for gene and cell therapies. Our RNA-based control systems will form part of the gene therapy cassette and enable precise control of therapeutic activity. Such control systems have huge potential to enhance both patient safety and therapeutic efficacy, for example by allowing a gene therapy to dynamically adjust its activity in response to patient disease state, recognise tissue type, or by allowing clinical control of the therapy after administration.

Cornell University is an American private Ivy League and land-grant doctoral university located in Ithaca, New York

Couragene believes that delivery of gene and biologic therapies should be safer and more efficient. Delivery of genome modification therapies to brain is further complicated by the physiological barriers. Couragene's STEP (Stimuli-responsive Traceless Engineering Platform) technology has demonstrated great potential to overcome the delivery hurdles for treatment of various diseases, including neurogenetic disorders.

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom.

CSL Behring is a biopharmaceutical company that manufactures plasma-derived and recombinant therapeutic products.COVID-19: CSL says it is working on a plasma-based therapy for patients with more severe forms of COVID-19.The company is part of the CSL family of companies. The parent company, CSL Limited is based in Melbourne, Australia, employs more than 30,000 people, and delivers its life-saving therapies to people in more than 100 countries.

Cure Genetics is an innovative gene therapy company focused on the application and development of viral delivery systems and gene editing systems. In response to the clinical needs of complex genetic diseases and refractory tumors, it has conducted extensive international cooperation and established a breakthrough product pipeline, striving to become a benchmark in the global gene therapy field. The company was founded in 2016 and is located in Suzhou Industrial Park. It brings together core talents from different fields at home and abroad to build a complete differentiated platform around the AAV virus delivery system and CRISPR gene editing system. Possess core independent intellectual property rights and create core competitive barriers. At the same time, the company has established a scientific committee composed of internationally renowned scientists and executives from multinational pharmaceutical companies to lay a solid foundation for technology commercialization.

Cure Rare Disease™ is developing advanced therapeutics for those who have been diagnosed with rare, genetic diseases that have no treatment or cures. Through partnerships and collaboration, our world-renowned researchers build life-saving therapeutics that are intended to stop or reverse the progression of disease.

DegenRx is a biotech company developing AAV-mediated gene therapy for the treatment of Alzheimer’s disease. Using Adeno-Associated-Viral (AAV) vectors we deliver genes in the brain that initiate the local production of therapeutic antibodies

DiNAQOR is a genetic medicine platform company pioneering early-stage drug development and manufacturing to advance promising gene therapies to the clinic. Our patent-protected, industry leading platform is designed to overcome the challenges and limitations of advanced gene therapy development by using proprietary engineered heart tissue technology (EHT), a novel Loco-Regional Perfusion system (LRP) and a fully integrated manufacturing solution. Our company is headquartered in the Greater Zurich Area, Switzerland, with additional presence in Laguna Hills, California and Hamburg, Germany.

The Duke University Clinical Laboratoriesemploy a wide range of practices; from high-throughput, highly automated testing in their hematology, chemistry and immunoassay laboratories, to specialized mass spectrometry and molecular based testing in many of their subspecialty labs, to direct patient care bytransfusion services physicians. In total last year, their 850 clinical laboratorians deliveredover 10million resultsto support the care of the many hundreds of thousands of patients who came to Duke.In additional to supporting the clinical mission of DUHS, theyalso support the other two missions of theiracademic health care system: teaching and research.

Dyno Therapeutics is pioneering an artificial intelligence (AI) powered approach to gene therapy.Using machine learning and quantitative high-throughput in vivo experimentation, they are inventing new ways to design gene vectors with a focus on cell-targeting capsid proteins from adeno-associated virus (AAV), the most widely-used vector for gene therapies.

EdiGene is a clinical-stage company focused on translating gene-editing technologies into transformative genetic medicines for patients with significant unmet medical needs. Anchored by its proprietary technologies of gene editing, bioinformatics, and high throughput genome-editing screening, EdiGene advances the pipeline on therapeutic platforms of LEAPER-based in vivo RNA base editing, ex vivo gene-editing hematopoietic stem cells, and ex vivo gene-editing T cells for allogeneic CAR-T.

At Editas Medicine, we are pioneering the possible. Our mission and commitment is to harness the power and potential of CRISPR gene editing to develop a robust pipeline of medicines for people living with serious diseases around the world. Our goal is to discover, develop, manufacture, and commercialize transformative, durable genomic medicines for many diseases.

Eikonoklastes Therapeutics is an early stage biopharmaceutical company focused on rapidly advancing innovative medicines for numerous areas of significant unmet clinical need, starting with neurodegenerative diseases. We are iconoclasts who are passionate about finding new and better ways to treat disease. Our first-in-class gene therapy moves beyond single defective gene replacement. It is a transformational, disease-modifying gene supplement therapy with a unique mechanism of action that provides extra copies of a critical gene to cause overexpression of a critical neurotrophic factor that is essential for neuromuscular signaling and function. Our initial focus is on Amyotrophic Lateral Sclerosis (ALS, Lou Gehrig’s Disease), a rare, yet debilitating nerve disease that causes progressive muscle degeneration and eventual death. An estimated 5,000 new patients in the U.S. are diagnosed each year with ALS, and there are few treatment options beyond supportive care. Our dynamic discovery and development approach allows us to focus on accelerated approvals and outsized returns for ALS and potentially other neurodegenerative diseases such as Parkinson’s, Huntington’s, Alzheimer’s, traumatic brain injury, and more.

ElevateBio is a technology-driven company built to power transformative genetic medicines today and for many decades to come. The Company commercializes its enabling technologies, manufacturing capabilities, and industry-leading expertise through partnerships to accelerate development across a breadth of therapeutic approaches and modalities. The ElevateBio ecosystem combines multiple R&D technology platforms – including Life Edit, a next-generation, full-spectrum gene editing platform; comprehensive cell engineering technologies; and an expanded viral and non-viral therapeutic delivery platform – with BaseCamp®, its end-to-end genetic medicine cGMP manufacturing and process development business, to accelerate the discovery and development of advanced therapeutics.

Eli Lilly and Company discovers, develops, and markets human pharmaceuticals worldwide. It offers Basaglar, Humalog, Humalog Mix 75/25, Humalog U-100, Humalog U-200, Humalog Mix 50/50, insulin lispro, insulin lispro protamine, insulin lispro mix 75/25, Humulin, Humulin 70/30, Humulin N, Humulin R, and Humulin U-500 for diabetes; and Jardiance, Trajenta, and Trulicity for type 2 diabetes. The company provides Alimta for non-small cell lung cancer (NSCLC) and malignant pleural mesothelioma; Cyramza for metastatic gastric cancer, gastro-esophageal junction adenocarcinoma, metastatic NSCLC, metastatic colorectal cancer, and hepatocellular carcinoma; Erbitux for colorectal cancers, and various head and neck cancers; Retevmo for metastatic NSCLC, medullary thyroid cancer, and thyroid cancer; Tyvyt for relapsed or refractory classic Hodgkin’s lymph and non-squamous NSCLC; and Verzenio for HR+, HER2- metastatic breast cancer, node positive, and early breast cancer. It offers Olumiant for rheumatoid arthritis; and Taltz for plaque psoriasis, psoriatic arthritis, ankylosing spondylitis, and non-radiographic axial spondylarthritis. The company offers Cymbalta for depressive disorder, diabetic peripheral neuropathic pain, generalized anxiety disorder, fibromyalgia, and chronic musculoskeletal pain; Emgality for migraine prevention and episodic cluster headache; and Zyprexa for schizophrenia, bipolar I disorder, and bipolar maintenance. Its Bamlanivimab and etesevimab, and Bebtelovimab for COVID-19; Cialis for erectile dysfunction and benign prostatic hyperplasia; and Forteo for osteoporosis. The company has collaborations with Incyte Corporation; Boehringer Ingelheim Pharmaceuticals, Inc.; AbCellera Biologics Inc.; Junshi Biosciences; Regor Therapeutics Group; Lycia Therapeutics, Inc.; Kumquat Biosciences Inc.; Entos Pharmaceuticals Inc.; and Foghorn Therapeutics Inc. Eli Lilly and Company was founded in 1876 and is headquartered in Indianapolis, Indiana.

Elpida Therapeutics is a biopharmaceutical company that focuses on developing treatments for rare diseases, offering hope to children and families affected.

Emendo Biotherapeutics develops next generation gene editing tools for genetic disorders, addressing the current technological gaps for realizing the promise of gene therapy. We leverage our expertise in protein engineering to bring innovative and disruptive protein tools to resolve the bottleneck of the field.

Emmune, Inc. is a start-up biotechnology company that has a novel approach for treating and preventing HIV infection. We engineered a synthetic antibody from the receptors that HIV uses to infect T cells, CD4 and CCR5. Because this synthetic antibody, known as “eCD4-Ig,” is built from the viral receptor and co-receptor, it recognizes 100% of HIV-1 isolates. A single intramuscular inoculation with an AAV vector expressing eCD4-Ig protected monkeys from multiple intravenous challenges with live virus.

Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation platform. The Encoded approach offers potentially unprecedented gene specificity and cell selectivity to unlock novel opportunities by targeting a range of disease mechanisms. Encoded’s technology is compatible with any delivery system to control where and when therapeutic transgenes are expressed, thereby shaping the functionality of target cells and holding broader therapeutic potential beyond CNS disorders.

Endsulin is a pre-clinical stage gene therapy company whose mission is to end insulin dependence for good and free people from being defined by diabetes. Our approach employs a widely studied and proven viral vector to deliver our patented genetic factor to the liver and restore the body’s own ability to produce and regulate insulin. With a single administration, Endsulin’s revolutionary genetic therapy for T1DM delivers a patented genetic factor that will “switch on” new insulin-producing cells to confer years – and perhaps a lifetime – of freedom from injecting artificial insulin, realizing the goal that has eluded researchers since the discovery of insulin. Founded by an award-winning transplant surgeon and world leader in diabetes care and supported by a team of experts in gene therapy, endocrinology, diabetes science, and pharmaceutical development, Endsulin has the scientific, medical, and commercial expertise to develop its innovative therapy quickly, filling an enormous unmet need and bringing us one step closer to ending insulin dependence for good — and for the good of everyone with T1DM.

Epic Bio is a biotechnology company that focuses on biomedical research and genetics.

Eppendorf is a leading life science company that develops and sells instruments, consumables, and services for liquid-, sample-, and cell handling in laboratories worldwide. Its product range includes pipettes and automated pipetting systems, dispensers, centrifuges, mixers, spectrometers, and DNA amplification equipment as well as ultra-low temperature freezers, fermentors, bioreactors, CO2 incubators, shakers, and cell manipulation systems. Consumables such as pipette tips, test tubes, microtiter plates, and disposable bioreactors complement the range of highest-quality premium products. Eppendorf products are most broadly used in academic and commercial research laboratories, e.g., in companies from the pharmaceutical and biotechnological as well as the chemical and food industries. They are also aimed at clinical and environmental analysis laboratories, forensics, and industrial laboratories performing process analysis, production, and quality assurance. Eppendorf was founded in Hamburg, Germany in 1945 and has about 5000 employees worldwide. The company has subsidiaries in 33 countries and is represented in all other markets by distributors. Imprint: www.eppendorf.com/imprint

We are ESTEVE, a global pharmaceutical company with one defined mission: improving lives. Founded in 1929 and headquartered in Barcelona, we have a strong international footprint, with affiliates across Spain, Portugal, Italy, Germany, France, the UK, and the USA. As a leader in speciality pharmaceuticals, we focus on creating highly specialized treatments that address significant unmet medical needs. Our expertise extends to Contract Manufacturing, where we produce Active Pharmaceutical Ingredients (APIs) through world-class facilities in Spain, Mexico, and China. Driven by our core values—people matter, transparency, and accountability—we’re committed to innovation, quality, and making a meaningful difference in healthcare. Because we care not only about what we do, but also about how we do it. #ESTEVE #AdvancingHealthTogether #Makeitsustainable #Pharmaceuticals #Innovation #SpecialtyPharma #APIs

Eurofins Lancaster Laboratories is one of the largest commercial contract laboratories in the world providing comprehensive laboratory services in the pharmaceutical, biopharmaceutical and environmental sciences, both in our laboratories and in our client facilities through our Professional Scientific Services (PSS) program. Our goal is to be the premier provider of laboratory services worldwide while delivering an outstanding service experience. We serve clients from a diverse range of businesses and industries including Fortune 100 Industrial companies, the world’s largest pharmaceutical/biopharmaceutical companies as well as virtual, small and mid-sized companies throughout the world. We also serve local and national governments. With facilities in Lancaster, Pennsylvania, Portage, Michigan, and Dungarvan, Ireland, we have a global capacity of more than 300,000 square feet. Our state-of-the-art facilities coupled with the highest level of instrument technology allow us to handle projects of virtually any scope and size.

Evox Therapeutics is harnessing and engineering the natural trafficking capabilities of extracellular vesicles to develop an entirely novel class of biotherapeutics. By combining groundbreaking exosome technology from well-renowned Oxford University and the Karolinska Institutet – the home of the Nobel Prize – we aim to revolutionise the treatment of a broad range of severe disease, with profound implications for human health. Based in Oxford, UK, Evox Therapeutics was founded in 2016 by Professor Matthew Wood of Oxford University, Assistant Professor Samir EL Andaloussi and Dr Per Lundin of the Karolinska Institutet, to capitalise on foundational intellectual property springing from the groundbreaking exosome research carried out in these two world-leading labs. Backed by Oxford Sciences Innovation and with a driven multidisciplinary team, Evox is building an exosome powerhouse focusing on the development of a modular platform for clinical translation of a broad range of exosome-based therapeutics. Evox Therapeutics has built a comprehensive intellectual property portfolio encompassing key aspects of EV-based nucleic acid and protein delivery technology. Coupled with targeting technology and proprietary manufacturing and purification methods, the company is set to develop transformational therapeutics across a wide range of disease areas, using an equally wide array of therapeutic modalities.

Excision BioTherapeutics is a private company focused on developing advanced gene editing therapeutics to address the medical need for the treatment of life-threatening disease caused by viral infections. Our mission is to advance gene editing therapeutics for our indications into safe and efficacious medicines that will greatly improve the lives of infected individuals around the world.

Exegenesis Bio is a clinical stage global gene therapy company with operations in Philadelphia, Boston and China. The company’s innovative gene therapy pipeline is based on proprietary capsids, promoters and unique protein engineering designs. Two programs have advanced to the clinical stage: (1) Type I Spinal Muscular Atrophy (SMA) AAV gene therapy in China (2) neurovascular AMD (wet AMD) rAAV gene therapy in USA The company has built state-of-the-art cGMP manufacturing facilities that include 500L and 2,000L disposable bioreactors for viral vectors and 30L disposable fermenters for plasmids. Exegenesis Bio has raised over $150 M since inception in 2019 and currently employs over 200 scientific and operations staff worldwide. Website: https://www.exegenesisbio.com/ Contacts: Company Information: Contact@ExegenesisBio.com Business Development: BD@ExegenesisBio.com Careers: Careers@ExegenesisBio.com

Exothera is a contract research, development, and manufacturing organization (CDMO) dedicated to viral vector and nucleic acids production. As a technology-driven company, Exothera provides best-in-class bioprocessing expertise that capitalizes on the best and most innovative manufacturing technologies developed within the Univercells group. The company delivers accelerated discovery services as well as process development/optimization to rapidly reach GMP clinical and commercial production of viral vectors and nucleic acids. At Exothera and Univercells, Our mission is to make life-changing therapies available to all. As a Univercells company, Exothera capitalizes on novel manufacturing technologies and best-in-class bioprocessing expertise to provide custom-made process optimization and GMP clinical and commercial production of viral vectors. Based on its extensive technology expertise, Exothera selects technologies to optimally answer customer needs for cost-effective and agile viral vector manufacturing. Company number: 0744643660 VAT: BE0744643660

We are a specialty CDMO for LV vector manufacture & use. Our three platforms: Expression Cassette Optimization (ECO), LentET (3rd gen system), & LentET Express (cGMP mfg.) enable GMP LV manufacture 10 months from sequence submittal. ECO increases expression up to 8 fold. LentET plasmids are ECO optimized, have improved safety, produce potent LVs & may be available royalty/licensing free depending upon the specifics. With full CMC & regulatory support, Expression is your preferred CDMO of choice.

Expression Therapeutics, Inc. is a fully integrated clinical stage gene and cell therapy company developing novel, potentially curative, therapeutics for hematology and oncology. The Company, based in Atlanta, Georgia, has a state-of-the-art 43,000 square foot manufacturing facility in West Chester, Ohio. Expression Therapeutics, Inc. also built a state-of-the-art scalable 43,000 square foot manufacturing facility in Cincinnati, Ohio.

Factor Bioscience develops new technologies in a little-explored area that we believed had enormous potential: messenger RNA ("mRNA").

Flagship Pioneering is a biotechnology company that invents and builds platform companies, each with the potential for multiple transformative human health and sustainability products. Since its launch in 2000, Flagship has originated and fostered more than 100 scientific ventures. The current Flagship ecosystem comprises over 40 companies, including Denali Therapeutics, Foghorn Therapeutics, Generate:Biomedicines, Inari, Indigo Agriculture, and Tessera Therapeutics.

Forge is a gene therapy development engine, focused on enabling access to life changing gene therapies and helping bring them from idea into reality. We partner with innovators in the gene therapy community: scientists, physicians, biotech/pharma companies, and patient groups. We bring expertise in gene therapy manufacturing and therapeutics development to the table, helping champion teams that are navigating the long road from the lab bench to the bedside. With a patients-first approach, we are forging new models for working together to better accelerate these transformative medicines to reach those who need them most.

Form empowers scientists to harness the data, computing power and collaboration that drive how today's breakthroughs are made.

Fortress Biotech, Inc. (“Fortress”) is an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue. The company has seven marketed prescription pharmaceutical products and over 20 programs in development at Fortress, at its majority-owned and majority-controlled partners and subsidiaries and at partners and subsidiaries it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market areas, including oncology, rare diseases and gene therapy, which allow it to create value for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is focused on leveraging its significant biopharmaceutical industry expertise and network to further expand the company’s portfolio of product opportunities. Fortress has established partnerships with some of the world’s leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including AstraZeneca, City of Hope, Fred Hutchinson Cancer Center, Nationwide Children’s Hospital and Sentynl.

Fractyl Health is a leader in creating innovative therapies to address the root cause of type 2 diabetes. We were founded based on groundbreaking research and biological insights showing the key role of the intestine in metabolic control. Our therapies are uniquely designed to target and control this controller for the treatment of metabolic disease. Our lead program, Revita DMR, is a first-in-class procedural therapy that is now in late-stage clinical trials for the treatment of type 2 diabetes. Our team is made up of passionate innovators at the intersection of biology and technology, working to deliver better real-world outcomes for people with metabolic disease.

Frontera is a leading clinical-stage biotechnology company that seeks to develop the best gene therapy medicines to improve the lives of patients across multiple disease areas. Frontera's APEX Technology & Manufacturing Platform is an innovative adeno-associated virus (AAV) gene expression system. It includes novel and clinically validated AAV vectors, efficient and fully-integrated CMC processes, and access to early clinical data, making it possible to develop novel gene therapy products with quicker speed-to-market and lower cost.

Fujifilm Cellular Dynamics, Inc. develops and manufactures biologically relevant human cells derived from induced pluripotent stem (iPS) cells. Our iCell® and donor-specific MyCell® Products are highly pure, highly reproducible, and available in industrial quantity to enable drug discovery, toxicity testing, stem cell banking, and cell therapy development.

At Fuse Vectors, our mission is to revolutionize AAV gene therapy development. Traditional cell-based technologies have hindered progress, posing challenges in safety, efficacy, development time, and cost. Our Cell-Free AAV technology surpasses these methods, producing safer, more effective, and cost-efficient AAV viral vectors in a fraction of the time.

GEMMABio, a new therapeutics company, will serve as the research and innovation arm to speed the research of and global access to life-changing advanced therapies for those living with rare diseases. Alongside Franklin Biolabs, GEMMABio will advance gene therapy research and deliver therapeutics to patients around the world significantly faster.

A Biotechnology Research private company. Mission is to take on one of the largest problems in all of medicine – the leading cause of disability, chronic pain, and a significant risk factor for opioid addiction – osteoarthritis (OA).

Gene Biotherapeutics is a biotechnology company that focuses on the clinical development and commercialization of DNA-based biotherapeutics for the treatment of cardiovascular disease.

GeneCraft is a pre-clinical-stage biotech company dedicated to improving treatment and clinical outcomes for patients through gene therapy that harness the power of the immune system. The company's technology allows for the development of gene therapies for the treatment of cancer.

Generation Bio Co., a genetic medicines company, develops gene therapies for the treatment of rare and prevalent diseases. The company is developing a portfolio of programs for rare and prevalent diseases of the liver and retina. It also focuses on the diseases of skeletal muscle, central nervous system, and oncology. The company was formerly known as Torus Therapeutics, Inc. and changed its name to Generation Bio Co. in November 2017. Generation Bio Co. was founded in 2016 and is headquartered in Cambridge, Massachusetts.

Gene Therapy Research Institution Co., Ltd. is headquartered in Japan. Based on extensive research, the company is committed to providing safe and effective gene therapies for refractory diseases for which effective therapies have not yet been established. Focusing on Parkinson's disease, amyotrophic lateral sclerosis (ALS) and Alzheimer's disease, the company strives to find safer viral vectors and more effective genetic technologies to bring new treatment options to patients.

Created in 1990 and funded by AFM-TELETHON, Genethon, a pioneer in deciphering the human genome and identifying the genes associated with genetic diseases is today fully dedicated to the design and development of gene therapy treatments for rare diseases. At Genethon more than 180 scientists, clinicians and engineers specialized in gene therapy drug development, from research to the clinic, have joined forces in order to provide these innovative treatments to patients affected with rare disorders. These treatments also pave the way for new therapeutic approaches for frequent disorders.

At GeneVentiv, we’re blazing a trail. We’re driven by curiosity. We’re fueled by learning expressed as innovation. Our mission is to discover, develop and deliver gene therapies for patients who had no hope of a cure – until now. We are founded on discovery bringing together insights into Adeno Associated Virus (AAV) directed gene therapy, Hemophilia and how to use the common pathway in the clotting cascade to surmount the obstacle of inhibitors. Patients with inhibitors are unable to respond to current therapies and gene therapies in development due to neutralizing antibodies (inhibitors) formed by the body in response to treatment with missing clotting factor. Our goal is to cure inhibitor patients with a single lifetime infusion, not to offer a half measure of weekly injections of a bypass agent.

Genflow Biosciences plc (LON:GENF) (OTCQB:GENFF) is a UK-based biotech company with R&D facilities in Belgium and a U.S. office in Cambridge, MA, driven by one mission: to deliver therapeutics that potentially halt or slow the ageing process in humans and dogs. The company’s lead compound works through the delivery of a centenarian variant of the SIRT6 gene and has yielded promising preclinical results. Managed by an experienced team with decades of experience in the pharmaceutical and biotechnology sectors, the company is optimistic that development programs will continue at pace in the next 24 months.

At Genix Cure, we're on a mission to redefine well-being through the power of nature. We craft innovative, natural remedies that promote health and vitality, utilizing the genesis of nature's ingredients. Our commitment to authenticity and purity ensures that our products can be consumed daily, fostering wellness without compromise. Join us on our journey to a healthier, more rejuvenated life, where we believe that the path to well-being begins with nature's own genesis.

Genkore is a Korea-based biotech company specializing in genetic scissors and gene therapy. They have developed a healthcare platform designed to provide gene therapy for medical emergencies that may occur in the future. The company has also announced collaborations with US-based companies for in vivo gene editing therapy and has signed deals worth up to $650M for gene editing tech. Genkore is also involved in plant biotechnology and has a strong focus on research and development.

GenoSafe is a CRO providing analytical testing services for the evaluation of quality, safety and efficacy of Gene and Cell therapy candidates including : - GLP biodistribution, shedding and immunogenicity studies ; - QC testing, such as viral titration, safety and potency/efficacy testing ; - GCP bioanalysis of patient samples, including immunogenicity, shedding and gene expression studies. GenoSafe brings more than 15 years of experience in the development, qualification and validation of key analytical methods for product and sample testing.

Genosera is revolutionizing the gene therapy field with its next generation gene therapy treatments that can help stop the progression of diseases and allow patients to regain their health. Founded in 2019, Genosera is a privately held, gene therapy biotechnology company located in Columbus, Ohio.

Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprex’s technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. Genprex’s oncology program utilizes its proprietary, non-viral ONCOPREX® Nanoparticle Delivery System, which the Company believes is the first systemic gene therapy delivery platform used for cancer in humans. ONCOPREX encapsulates the gene-expressing plasmids using lipid nanoparticles. The resultant product is administered intravenously, where it is then taken up by tumor cells that express tumor suppressor proteins that are deficient in the body. The Company’s lead product candidate, REQORSA® (quaratusugene ozeplasmid), is being evaluated in three clinical trials as a treatment for non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC). Both NSCLC clinical programs received a Fast Track Designation from the Food and Drug Administration. Genprex’s diabetes gene therapy approach is comprised of a novel infusion process that uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes directly to the pancreas. In models of Type 1 diabetes, GPX-002 transforms alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body’s immune system. In a similar approach, GPX-003 for Type 2 diabetes, where autoimmunity is not at play, is believed to rejuvenate and replenish exhausted beta cells.

GenSight Biologics is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics for retinitis pigmentosa, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics’ lead product candidate, GS010, is in Phase III trials in Leber’s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible low vision and legal blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

Gilead Sciences, Inc., a research-based biopharmaceutical company, discovers, develops, and commercializes medicines in the areas of unmet medical need in the United States, Europe, and internationally. The company provides Biktarvy, Genvoya, Descovy, Odefsey, Truvada, Complera/ Eviplera, Stribild, and Atripla products for the treatment of human immunodeficiency virus (HIV) infection; Veklury, an injection for intravenous use, for the treatment of coronavirus disease 2019; and Epclusa, Harvoni, Vosevi, Vemlidy, and Viread for the treatment of liver diseases. It also offers Yescarta, Tecartus, Trodelvy, and Zydelig products for the treatment of hematology, oncology, and cell therapy patients. In addition, the company provides Letairis, an oral formulation for the treatment of pulmonary arterial hypertension; Ranexa, an oral formulation for the treatment of chronic angina; and AmBisome, a liposomal formulation for the treatment of serious invasive fungal infections. Gilead Sciences, Inc. has collaboration agreements with Arcus Biosciences, Inc.; Pionyr; Tizona; Tango Therapeutics, Inc.; Jounce Therapeutics, Inc.; Galapagos; Janssen; Japan Tobacco, Inc.; Gadeta; Bristol-Myers Squibb Company; Merck; and Novo Nordisk A/S. The company was founded in 1987 and is headquartered in Foster City, California.

GQ Bio is pioneering a high-capacity gene therapy vector platform (HCAd) that addresses some of the big challenges in the gene therapy field: Transfer of large and multiple genes with a single vector, highly efficient gene delivery (high transduction efficiency), and large-scale manufacturability. Based on its HCAd vector platform, GQ Bio develops transformative treatments for chronic, prevalent conditions such as osteoarthritis and intervertebral disc degeneration. GQ Bio is headquartered in Hamburg, Germany and has sites at Luckenwalde (greater Berlin area), Germany and Liège, Belgium.

Greffex™ is a privately held, multi-national company, headquartered in Aurora, Colorado. Established in 1999, Greffex has developed a novel genetic platform for the development and production of vaccines. Our proprietary GREVAX™ Universal Platform delivers vaccines in a faster, more affordable and more efficient manner. Once a pathogen has been identified, Greffex's team designs and produces a GREVAX™ vaccine in as little as 4 weeks. The state-of-the-art flexibility of the GREVAX™ Universal Platform makes it the ideal delivery vehicle for vaccines against a wide variety of infectious disease, including but not limited to Pandemic Influenza strains, Anthrax and Dengue Fever, as well as Malaria, Hepatitis C, Respiratory Syncytial Virus (RSV), Ebola, HPV, Plague, Tuberculosis and a host of other vaccine candidates.

Gritgen Therapeutics (here in after designated as "Gritgen") was founded in China and is a biotechnology company focused on gene therapy. They have launched a commercial GMP facility in China and have opened a good manufacturing practice (GMP) facility in Suzhou Industrial Park. Gritgen provides an important vector foundation for the company to realize gene therapy for hemophilia A. The company has also launched a Phase 1 clinical trial to assess the safety of gene therapy for hemophilia A.

Gritstone Oncology, Inc., an immuno-oncology company, engages in developing tumor-specific cancer immunotherapies to fight various cancer types. Its lead product candidate is GRANITE, which is in Phase I/II clinical trial for the treatment of solid tumors, including metastatic non-small cell lung cancer, as well as gastroesophageal, bladder and microsatellite stable, and colorectal cancers. The company is also developing SLATE, an off-the-shelf immunotherapy candidate which is in Phase I/2 clinical study for the treatment of common solid tumors, including metastatic non-small cell lung cancer, colorectal cancer, pancreatic cancer, and other mutation-positive tumors. Gritstone Oncology, Inc. has a strategic collaboration with bluebird bio, Inc. The company was founded in 2015 and is headquartered in Emeryville, California.

Discovering and developing innovative medicines to preserve people’s sight and fight the devastating impact of blindness around the world.

Harvard Bioscience is a global developer, manufacturer and marketer of a broad range of specialized products, primarily apparatus and scientific instruments used to advance life science research at pharmaceutical and biotechnology companies, universities and government laboratories worldwide. We sell our products to thousands of researchers in over 100 countries through our full-line catalog (and various other specialty catalogs), our websites, and through distributors, including GE Healthcare, Thermo Fisher Scientific Inc., and VWR. We have sales and manufacturing operations in the United States, the United Kingdom, Germany, and Spain and sales facilities in France and Canada.

Heidelberg University is a higher education institution offering a variety of academic programs and research opportunities.

More than six hundred years ago, the first German university was founded in Heidelberg. Today, it attracts international acclaim for its life sciences – especially for medicine. As one of Europe’s largest and most modern medical centers, Heidelberg University Hospital’s 46 specialist departments offer medical care of the highest international standards in all areas of specialty with a focus on oncology care and on other diseases that require complex treatments. Heidelberg University Hospital’s active collaboration with national research facilities attracts patients from many countries worldwide.

Helixmith is a gene therapy company headquartered in Seoul, Korea, developing new and innovative biopharmaceuticals for previously untreated diseases, and is listed on KOSDAQ. The company has an extensive gene therapy pipeline, including a CAR-T program targeting several different types of solid tumors and an AAV vector program targeting neuromuscular diseases. Engensis (VM202), a more advanced pipeline candidate, is a plasmid DNA therapy being studied for DPN, diabetic foot ulcers, claudication, amyotrophic lateral sclerosis (Phase 2 beginning in late 2020), coronary artery disease and Charcot-Marie-Tooth disease. Helixmith clinical development activities are based in San Diego, California,

Hengyu Biotechnology is a CXO platform founded by a scientific research team that provides biosafety testing and related process validation. It is a comprehensive provider founded by a group of top scientists, dedicated to providing innovative solutions in the biotech and biopharma space.

The Faculty of the Horae Gene Therapy Center is dedicated to developing therapeutic approaches for rare inherited disease for which there are no cures. They utilize state of the art technologies to correct the genetic mutations underlying the diseases. The Center focuses on AAT Deficiency, Amyotrophic Lateral Sclerosis (Lou Gehrig's Disease), Canavan Disease, Cystic Fibrosis, Tay-Sachs & Sandoff diseases, Retinitis Pigmentosa, Huntington's disease, Hypercholesterimia, and Cardiac Arrhythmia.The Center operates under the umbrella of the Advanced Therapeutics Cluster (ATC) of UMass, whose mission is to apply knowledge flowing from the latest biomedical discoveries, develop new ways to treat diseases, and drive those therapies into clinical trials.

HuidaGene Biotechnology Co., Ltd (辉大基因) is a global biotechnology company focusing on the discovering, engineering, and developing CRISPR-based genetic medicine to rewrite the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene is committed to addressing patient's needs globally with various preclinical therapeutic programs covering ophthalmology, otology, myology, and neurology. Company's CRISPR-based therapeutics offer the potential to cure patients with life-threatening conditions by repairing the cause of their disease. We are committed to transform the future of genome-editing medicine.

Headquartered in Cambridge, Massachusetts, Hunterian Medicine LLC is a gene-editing company working to cure genetic diseases by correcting DNA mutations using its innovative CRISPR delivery technology. The company’s patented technology solves the problem of CRISPR delivery with a “2-for-1” genetic element that enables delivery via a single adeno-associated virus (AAV).

Headquartered in North Bethesda, Maryland, Ibex Biosciences, Inc. ("Ibex") distinguishes itself in the early-stage biotech sector with its varied research and development approach. ​Ibex boasts a diverse portfolio that includes therapeutic antibodies, gene therapies, and regenerative medicine programs. We are proud of our proven capacity to discover and validate potential therapies from scratch, thus eliminating the need for external technology acquisitions or in-licensing. Our accomplishments are underscored by a series of in-vivo studies, which have delivered remarkable efficacy against a range of challenging medical conditions.

The International Centre for Genetic Engineering and Biotechnology ICGEB is an intergovernmental research organisation that operates within the United Nations common system. It was established in 1983 as a Centre for excellence in research, training and technology transfer to industry in the field of biotechnology to promote sustainable global development. With over 60 Member States and 40 Affiliated Centres worldwide, it runs 46 research programmes in its own laboratories in Trieste, Italy, New Delhi, India and Cape Town, South Africa. Almost 700 people work at ICGEB comprising 550 scientific personnel. The organisation has generated almost 3000 scientific publications in the Life Sciences since 1988 and currently holds over 80 active research grants. ICGEB has awarded almost 1400 fellowships, with over 300 fellows and 150 PhD students currently on board. It has organised over 560 Meetings and Courses worldwide and has funded over 550 Collaborative Research Programme-Research Grants. It has produced and distributed over 600 movies (podcasts) of high-scientific content in 50 topics in the Life Sciences - that are freely available for download on iTunes, YouTube and on the ICGEB Web site. Active in Public Engagement and on Social Media networks, ICGEB invites, in particular, its Alumni to join the ICGEB Alumni Group on Linkedin. http://www.icgeb.org

iECURE is a clinical-stage biotechnology company focused on delivering cures and transforming the lives of patients with devastating liver disorders, utilizing new advancements in genetic medicine. We are advancing our pipeline in close partnership with the world-class translational engine at the University of Pennsylvania’s Gene Therapy Program. Using in vivo editing, our methods focus on inserting functioning genes into patients’ genomes, which offers long-term, stable expression of those genes. With our team’s proven track record, as well as the University of Pennsylvania’s deep expertise and translational genetic medicine engine, reversing the course of these devastating diseases is now within reach.

Imanis Life Sciences has an unwavering commitment to the advancement of science. For many companies, profitability rules decision making. But at Imanis, leading scientific breakthroughs is the primary focus. We are about making a difference in the world by putting people in a better position than they were yesterday. We strive to do this by being a leader in laboratory assays and research services to accelerate the development of a broad range of next-generation therapies.

ImmunityBio (previously NantKwest (NASDAQ: NK)) is a pioneering, next generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and inflammatory diseases. NK cells are the body’s first line of defense due to the innate ability of NK cells to rapidly identify and destroy cells under stress, such as cancer or virally-infected cells. ImmunityBio is uniquely positioned to implement precision cancer medicine, with the potential to change the current paradigm of cancer care. Natural Killer cells are ancient cells in the human body designed to recognize and detect cells under stress or infected. Our “off-the-shelf” activated Natural Killer (NK) platform have the capacity to destroy cancer and virally infected cells from the body. The safety of our NK cells as well as their activity against a broad range of cancers have been tested in multiple phase 1 clinical trials in the United States, Canada and Europe. In addition to our NK cells capability to be administered in the outpatient setting as an “off-the-shelf” living drug, it serves as a universal cell-based therapy without need for individualized patient matching. Moreover, our NK cell based platform has been bioengineered to incorporate chimeric antigen receptors (CARs) and antibody receptors to further optimize targeting and potency in the therapeutic disease. Our targeted therapeutic areas include: (1) cancer, focusing on bulky hematological cancers and solid tumors as well as cancer stem cells, (2) infectious diseases, including viral, fungal and bacterial infections, and (3) inflammatory diseases, ranging from rare inherited diseases to more prevalent autoimmune disorders.

Indiana University is a Higher Education institution that provides academic programs and conducts research.

Innovative NeuroTechnologies, Inc. (INT) is a development-stage biotechnology company operating in “virtual mode” that is currently pursuing a pipeline of novel biologically-based and disease-modifying therapeutic solutions to prevent, delay, or mitigate the underlying dementia or cognitive decline associated with a spectrum of neurodegenerative disorders referred to as tauopathies, which includes: • Alzheimer’s disease (AD), • Frontotemporal dementia (FTD or Pick’s disease), • Chronic traumatic encephalopathy (CTE) or dementia pugilistica, • Progressive supranuclear palsy (PSP), • Corticobasal degeneration (CBD), • Familial form of frontotemporal dementia and parkinsonism linked to chromosome 17 (FTDP-17), and • Parkinson's disease dementia (PDD). The pathology of these seemingly disparate conditions is similar in that they involve dysregulation of the Tau protein and development of neurofibrillary tangles (NFTs), synaptic dysfunction, and/or neuronal loss, which underlie the observed dementia or cognitive deficits. Unfortunately, there are no effective therapies presently available for this spectrum of disorders, and neurodegenerative tauopathies remain a very significant socioeconomic burden and tremendous unmet medical need worldwide.

Intellia Therapeutics is a leading genome editing company whose mission is to develop potentially curative gene editing treatments that can positively transform the lives of people living with severe and life-threatening diseases. We are focused on the development of proprietary therapeutics using a recently developed biological tool known as the CRISPR/Cas9 system. The promise of the CRISPR/Cas9 system is the driving force behind the creation of Intellia. Our founders have a shared belief that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. This technology offers the potential for us to develop curative therapeutic options for patients with chronic diseases by addressing the underlying cause of the disease. It can bring new hope and cures to people who now have nowhere to turn for help. Our combination of deep scientific expertise and clinical development experience, along with our leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Intellia has the right people, assets and ambitious vision needed to take full advantage of the CRISPR/Cas9 technology’s attributes – high potency, specificity, simplicity of use, broad applicability, and multifunctional programmability – and accelerate the advancement of curative products into the clinic. View our social media community guidelines at https://www.intelliatx.com/terms-and-conditions/ .

Intima Bioscience - A clinical stage gene and cell therapy company focused on curative intent in solid tumor cancer

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy. It is involved in developing neurology products that include Tominersen for Huntington’s diseases; Tofersen for amyotrophic lateral sclerosis (ALS); IONIS-MAPTRx for Alzheimer's diseases; IONIS-C9Rx for genetic form of ALS; ION859 for Parkinson’s disease; ION464 for multiple system atrophy and Parkinson’s disease; ION541 for amyotrophic lateral sclerosis; and IONIS-DNM2-2.5Rx for centronuclear myopathy. The company is also developing products for cardiometabolic disease, such as IONIS-TTR-LRx for transthyretin amyloidosis and IONIS-GHR-LRx for acromegaly; IONIS-APOCIII-LRx for familial chylomicronemia syndrome and TG- driven diseases; IONIS-APOCIII-LRx for treatment resistant hypertension; Pelacarsen for Lp(a)cardiovascular disease risk reduction; Vupanorsen for sHTG/CVDRR; IONIS-FXI-LRx for clotting disorders; ION449 for cardiovascular diseases; and IONIS-GCGRRx for diabetics. In addition, it develops IONIS-ENAC-2.5Rx for cystic fibrosis and chronic obstructive pulmonary disease; IONIS-PKK-LRx for hereditary angioedema; Danvatirsen for cancer; IONIS-TMPRSS6-LRx for beta-thalassemia and iron toxicity; IONIS-HBVRx for hepatitis B virus; IONIS-AR-2.5Rx for prostate cancer; IONIS-FB-LRx for age-related macular degeneration and IgA nephropathy; and ION357 for retinitis pigmentosa. It has a collaboration agreement with AstraZeneca; GlaxoSmithKline plc; Janssen Biotech, Inc.; Bayer AG; Novartis AG; Pfizer, Inc.; Biogen Inc.; and Roche. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.

Isolere's mission is to increase global access to life-changing therapeutics and vaccines by offering novel purification reagents to alleviate manufacturing bottlenecks. The IsoTag™ reagents reimagine bioprocessing by combining target specific affinity ligands with phase separating biopolymers. The Affinity Liquid Phase Separation (ALPS) allows target biologics to be separated on the basis of affinity and size in a single process and can be implemented in a variety of techniques including microfiltration TFF and centrifugation processes. Isolere is currently developing purification solutions for adeno-associated virus (AAV), lentivirus (LV), adenovirus (AdV) and mRNA. Visit our website to learn more! Isolere Bio was acquired by Donaldson Company, Inc. (NYSE: DCI), a leading worldwide provider of innovative filtration products and solutions, in February 2023.

Created in 2005 through a collaboration between Inserm – Institut National Health and Medical Research – and AFM-Telethon – French Association against Myopathies – I-Stem is the largest French laboratory research and development dedicated to human pluripotent stem cells, embryonic origin or obtained by reprogramming gene. I-Stem is part of the Biotherapy Institute for Rare Diseases, which includes so far the four centers of research and development funded directly by the AFM Telethon. The specific vocation of I-Stem is to explore all the therapeutic potential of human pluripotent stem cells for applications in patients affected by rare diseases of genetic origin. In this context, our teams are developing two major areas of research. The first one is cell therapy, which aims to replace lost or diseased cells to other cells with the same characteristics, produced in the laboratory from pluripotent stem cells. The second area is pharmacology based on automated screening of large libraries of compounds with therapeutic potential, following modeling of molecular mechanisms associated with diseases, as revealed by the study of pluripotent stem cells from affected donors. I-Stem teams are currently working on a dozen genetic diseases that affect different organs. The Institute also hosts every year many researchers interested in other diseases and provides training and technology support.

Jaguar Gene Therapy, LLC is dedicated to accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable patient populations. The company is made up of a proven team of experts with unparalleled CMC (Chemistry, Manufacturing and Controls), regulatory, clinical and commercial acumen who have first-hand experience in bringing novel gene therapy treatments to patients and their families. Committed to patient safety and product purity, Jaguar is rapidly advancing an initial pipeline of three programs targeting: 1) Type 1 galactosemia; 2) a genetic cause of autism spectrum disorder and Phelan-McDermid syndrome and other severe neurodevelopmental disorders with a SHANK3 mutation or deletion; and 3) Type 1 diabetes. The company continues to evaluate opportunities to expand its pipeline using the strength of the team and close relationships with numerous academic institutions.

At Janssen, we never stop working toward a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, and you can count on us to keep working tirelessly to make that future a reality for patients everywhere, by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Arterial Hypertension. Visit us: www.janssen.com Follow us: @JanssenGlobal on Twitter and Instagram Watch our videos on our YouTube channel: http://bit.ly/1LY2xQ5

Johns Hopkins University is a higher education institution known for its research initiatives and academic programs.

At Johnson & Johnson, we believe good health is the foundation of vibrant lives, thriving communities and forward progress. That’s why for more than 135 years, we have aimed to keep people well at every age and every stage of life. Today, as the world’s largest and most broadly-based health care company, we are committed to using our reach and size for good. We strive to improve access and affordability, create healthier communities, and put a healthy mind, body and environment within reach of everyone, everywhere. We are blending our heart, science and ingenuity to profoundly change the trajectory of health for humanity.

KaliVir Immunotherapeutics was founded in 2019 to pioneer a unique and novel approach to cancer treatment that engages a virus' natural ability to replicate in and kill cancer cells. This modality is known as oncolytic viral immunotherapy. KaliVir's cutting-edge, next generation oncolytic viral immunotherapy programs combine technologies developed in-house and licensed from University of Pittsburgh's world-class oncolytic virus and immunotherapy research programs.

Kinea Bio is a patient-focused biotechnology company striving to accelerate the development of next-generation AAV-based gene therapies to treat various diseases. The company is located in Seattle, Washington and is dedicated to developing innovations to fight genetic diseases.

At Kite, a Gilead Company, our singular focus is cell therapy to treat and potentially cure cancer. Our goal is to bring the promise of cell therapy to as many eligible patients as possible who may benefit. Since 2009, Kite has been at the forefront of cancer immunotherapy. Today, we are proud to be the leader in engineered T cell therapy for hematologic malignancies, but we recognize that this is just the beginning. Patients are at the heart of our focus on the cure, which is why we are committed to pursuing cell therapy as a transformative treatment option for as many people with cancer as possible who may benefit. Cancer is personal to us. Unlike traditional pharmaceutical manufacturing, our therapies are not created on an assembly line. Cell therapy specialists at our manufacturing facilities reengineer a patient’s own immune cells to attack and fight their cancer cells — one patient at a time. That sense of connection to the people we serve motivates us daily at Kite. Our corporate headquarters are located in Santa Monica, California, and we have dedicated cell therapy manufacturing facilities in California, Maryland and the Netherlands. If you’re passionate about changing the future of cancer treatment, let’s talk.

Klotho Neurosciences is dedicated to realizing the potential of biologic, cell and gene therapies to offer transformative patient outcomes in areas of high unmet medical need by extending the reach of protein, cell, and gene therapies to highly prevalent neurodegenerative disorders like Alzheimer’s, amyotrophic lateral sclerosis (ALS), Parkinson’s, and other age-related disorders. Our vision is to build a leading gene therapy company for the treatment of neurodegenerative diseases by progressing our patented á-Klotho gene therapy research programs and identifying, developing, and commercializing other novel gene therapy treatments and in vitro diagnostics for neurodegenerative diseases, and other age-related pathologies. We have assembled a portfolio of gene therapy candidates in partnership with leading scientific institutions and have built a team with extensive experience in the biotechnology commercialization and gene therapy space. Our team will pursue new innovations in vector design and delivery to optimize our investigational gene therapy products for safety, potency, durability, and clinical response. We plan on building integrated internal development capabilities from product development through commercialization and focus on accelerating the pace of product development in the clinic. In addition, as part of our ongoing business strategy, we continue to explore potential opportunities to acquire or license new product candidates as well as opportunities for partnership or collaboration on our existing products in development.

Kriya Therapeutics, Inc. is a biopharmaceutical company developing gene therapies for diseases affecting millions of people around the world. With operations in Palo Alto, California and Research Triangle Park, North Carolina, Kriya has raised over $450 million to advance a broad pipeline of gene therapies for ophthalmology, neurology, and metabolic diseases.

Kriya Therapeutics, Inc. is a biopharmaceutical company developing gene therapies for diseases affecting millions of people around the world. With operations in Palo Alto, California and Research Triangle Park, North Carolina, Kriya has raised over $450 million to advance a broad pipeline of gene therapies for ophthalmology, neurology, and metabolic diseases.

At Latus Bio, we are developing novel therapeutics for CNS disorders with our revolutionary AAV capsids. We have developed capsid variants with unprecedented potency, specificity, and trophism. All of our capsids were designed with a particular disease in mind, in order to find the right capsid and the right route of administration to target the most relevant brain regions and cell types for each specific indication. Founded on the groundbreaking work of Professor Beverly Davidson from the Children's Hospital of Philadelphia, Latus Bio is committed to transforming patient care through targeted, potent, and safe gene therapies. We are focused on CNS diseases such as CLN2 (Batten Disease) and Huntington’s Disease, aiming to fundamentally alter the course of these challenging conditions by always putting the patients first. We are not just developing therapies; we are revolutionizing the future of healthcare with innovations that reduce therapeutic load, enhance safety, facilitate manufacturing, and expand access. Latus Bio is powered by a diverse team of visionary scientists, clinicians, and industry leaders dedicated to tackling some of the most complex neurological disorders. With a robust pipeline and a strategy to enter clinical trials by late 2025, we are poised to make significant strides quickly. Be part of this transformative movement at Latus Bio. Let's unlock the potential of gene therapy to bring hope and healing to millions around the globe. Learn more about our mission, our people, and our promise at www.latusbio.com and connect with us on LinkedIn to follow our progress.

Based in New York City, Lexeo Therapeutics is a clinical-stage genetic medicines company dedicated to transforming healthcare by applying pioneering science to fundamentally change how disease is treated. Building on groundbreaking research from Weill Cornell Medicine and the University of California San Diego, Lexeo partners with preeminent institutions on the cutting edge of gene therapy research. Using a stepwise development approach, Lexeo is leveraging early proof-of-concept functional and biomarker data to advance a pipeline of cardiovascular and APOE4 associated Alzheimer's disease programs, and is led by pioneers and experts with decades of collective experience in genetic medicines, rare disease drug development, manufacturing and commercialization. For more information, please visit www.lexeotx.com.

LigaTrap has developeda patented portfolio of affinity ligands that can be used to purifymonoclonal and polyclonal immunoglobulins from various species.

Next generation CRISPR technology for programmed cell death. Focused on developing therapies for infectious disease and the microbiome. Locus Biosciences’ novel approach to precision antimicrobials works by taking advantage of a part of an immune system present in many bacteria called the CRISPR-Cas system. The CRISPR-Cas system protects bacteria from invaders such as viruses by creating small strands of RNA called CRISPR RNAs, which match DNA sequences specific to a given invader. When the CRISPR RNAs find a match, they activate Cas proteins that cut the DNA.

LOKON PHARMA is a Swedish biotech company in the forefront of developing immunostimulatory gene therapy for cancer-based on oncolytic viruses. Innovative gene engineering, solid expertise in cancer biology and immunology, as well as a genuine curiosity in life science expand Lokon’s pipeline towards groundbreaking new therapeutics for patients with high unmet medical need.

The comapanies' research and development efforts are focused exclusively on rare genetic neurodevelopmental disorders, a group of serious diseases with few or no treatment options.

Mammoth Biosciences is a biotechnology company focused on leveraging its proprietary ultracompact CRISPR systems to develop long-term curative therapies, as well as other applications such as decentralized precision diagnostics. Founded by CRISPR pioneer and Nobel laureate Jennifer Doudna and Trevor Martin, Janice Chen, and Lucas Harrington, the company’s ultracompact proteins are designed to enable in vivo gene editing in difficult to reach tissues utilizing both nuclease applications and new editing modalities beyond double stranded breaks, including base editing, gene writing, and epigenetic editing. The company is building out its pipeline of potential in vivo gene editing therapeutics and capabilities and has partnerships with leading pharmaceutical and biotechnology companies to broaden the reach of its innovative and proprietary technology. Mammoth’s deep science and industry experience, along with a robust and differentiated intellectual property portfolio, have enabled the company to further its mission to transform the lives of patients and deliver on the full promise of CRISPR technologies.

Mass Eye and Ear in Boston is a Harvard teaching hospital dedicated to eye (ophthalmology) and ear, nose, throat, head and neck (ENT) care and research.

Maze Therapeutics is translating novel genetic insights into lifesaving medicines, through an approach that integrates human genetics and functional genomics. Advances in these areas have enabled us to decode the mysteries of genetic modifiers in a range of severe diseases, which we aim to turn into medicines that mimic protective mutations. We have assembled a world-class team that we need to realize this vision, including creative scientific pioneers in genetics, experienced company builders, and biotech industry leaders.

Medigene AG (FSE: MDG1) is a leading immuno-oncology platform company dedicated to developing T-cell therapies to effectively eliminate cancer. Its end-to-end technology platform is built on multiple proprietary exclusive product development and product enhancement technologies, and allows Medigene to create best-in-class differentiated, T cell receptor engineered T cell (TCR-T) therapies for multiple solid tumor indications that are optimized for both safety and efficacy. This platform provides product candidates for both its in-house therapeutics pipeline and partnering.

MediSieve is a London biotech start-up developing Magnetic Haemofiltration, a revolutionary platform therapy that enables the physical removal of specific substances from the bloodstream of patients. It can be used (1) as a direct treatment, (2) to increase the safety and efficacy of other therapies, or (3) to enable personalised medicine. A unique, lifesaving and often cost-saving solution, there are several multi-billion-dollar markets available for this product in oncology, infectious diseases, auto-immune diseases, poisoning, drug overdose and others. Magnetic Haemofiltration has already been shown to rapidly, specifically, and effectively remove IL-6 from human plasma. The technology has completed pre-clinical validation, including animal studies, and is expected to start clinical studies in Q2 2022. MediSieve has raised £4M in equity funding and over £4M in non-dilutive grants. Seeking a further £8M at Series A to support pivotal clinical trials and regulatory approvals. EIS eligible. The company has been featured in numerous online and print media articles, including the Sunday Telegraph, Daily Mail, and Wired Magazine, and been winner or finalist in several start-up competitions such as Pitch@Palace, MassChallenge, OBN Awards, TCT Magazine Awards and CMS Healthcare Startup of the Year. Recently, our founder George Frodsham was awarded BBSRC Innovator of the Year and MIT Innovators Under 35.

Megadalton Solutions is a technology company that specializes in charge detection mass spectrometry for the characterization of large molecules, helping clients quantify AAV fractions and troubleshoot large molecule formulations.

MeiraGTx is a clinical-stage gene therapy company focused on developing potentially curative treatments for patients living with serious diseases.

Gene editing is changing the world. Founded by pioneers in the CRISPR/CAS revolution, Metagenomi is discovering the next generation of gene-editing systems for use in treating genetic diseases.

OUR MISSION: Deliver Innovation. Empower Discovery. Improve Life. For over 25 years, Mirus Bio has pioneered the development of transfection reagents and proprietary technologies for nucleic acid delivery applications. Our work is both technical and cutting-edge, and we promote a culture of creativity, responsibility, and respect. Our team members are engaged and motivated by the desire to support our customers and partners to achieve strong results. Mirus, together with Gamma Biosciences, is establishing the future of science and medicine by developing products that enable product developers within the cell and gene therapy space. As we approach a new era of addressing and conquering life-altering diseases, we will continue to expand our expertise and leadership in transfection to be integral in the future of advanced therapies.

Modalis Therapeutics Corporation (Tokyo Mothers 4883) is developing precision genetic medicines through epigenetic gene modulation. Modalis is advancing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM technology which enables the locus specific modulation of gene expression or histone modification without the need for double- stranded DNA cleavage, gene editing or base editing. Modalis is headquartered in Tokyo with laboratories and R&D facilities in Cambridge,

Mogrify® has developed a proprietary suite of platform technologies that utilize a systematic big-data approach to direct cellular reprogramming (Rackham et al., Nature Genetics, 2016) and the maintenance of cell identity (Kamaraj et al., Cell Systems, 2020). The platforms, MOGRIFY® and epiMOGRIFY®, developed over a 12-year period via a multi-national research collaboration, deploy next-generation sequencing, gene regulatory and epigenetic network data to enable the prediction of the transcription factors and growth factors required to produce any target human cell type from any source human cell type. Mogrify is applying its proprietary and award-winning platforms to generate the functional cell types required to transform the lives of patients, by delivering a novel class of in vivo reprogramming therapies across ophthalmology, otology, metabolic and other areas of degenerative disease. Uniquely positioned to address a regenerative medicine market estimated to be worth $150 billion USD by 2028, Mogrify is commercializing its technology via a combination of in vivo reprogramming therapy development, co-development partnerships, as well as the exploitation of the platform in other therapeutic and non-therapeutic applications. Based in Cambridge, UK, the Company has raised over $40 million USD funding from Parkwalk, Ahren Innovation Capital, 24Haymarket, Trend Investment Group, Dr. Darrin M. Disley, OBE, Dr. Jonathan Milner and the University of Bristol Enterprise Fund III, as well as strategic investors; Astellas Venture Management.

Myosana Therapeutics, Inc. is leading the efforts in developing new gene therapies that will slow skeletal muscle degeneration and heart failure.

Myrtelle Inc. is a patient-centered, innovation-driven gene therapy company transforming the treatment of neurological diseases. Our mission is to develop and deliver novel treatments for devastating disorders of the central nervous system, beginning with our lead program, gene therapy treatment for Canavan disease. Myrtelle’s social media channels are places for community members to connect and learn about Canavan disease and Myrtelle initiatives. Discussion of medical and health topics should never be construed as medical advice. Contact a health care professional in your area for personal medical advice. Myrtelle Inc. does not represent that the information on our social media channels is accurate, complete, reliable, useful, timely, or current and Myrtelle does not undertake an obligation to update any such information. Myrtelle does not endorse any opinions or statements expressed by others on our social media channels. Myrtelle does not expressly endorse opinions or statements expressed by individuals, businesses, or channels we may ‘follow’, ‘like’, or ‘share’. At times, Myrtelle may link to external websites. Myrtelle is not responsible for the content or policies of these websites. Content submitted to our social media channels is public and will not place Myrtelle under any obligation to you.

NanoCell Therapeutics is a privately-held biotechnology company with offices in King of Prussia, Pennsylvania, and Utrecht, the Netherlands. Our mission is to revolutionize in-vivo cell engineering through our groundbreaking non-viral, DNA-based gene therapy platform, primarily focusing on oncology and autoimmune diseases. At NanoCell, we’re driving innovation by transforming traditional adoptive cell therapy methods into advanced in-vivo treatments. Our ultimate goal is to simplify the treatment process, increasing patient accessibility, potential clinical benefits, and cost-effective manufacturing. We work both autonomously and in collaboration with partners to make substantial advancements in our targeted disease areas. For more information, visit http://www.nanocelltx.com).

Nanoscope is focused on proprietary ambient light activated optogenetic therapy to restore vision in people suffering from all forms of Retinitis Pigmentosa.

NanoVation Therapeutics is a pioneer in the design and creation of lipid nanoparticles (LNPs) for nucleic acid delivery. The company’s LNP technologies aim to overcome the limitations of existing nucleic acid delivery approaches, including the ability to target tissues outside the liver (extrahepatic delivery). NanoVation’s lead technology is its proprietary long-circulating LNP (lcLNP™) platform, which enables functional nucleic acid delivery to extrahepatic cell types and has demonstrated an improved therapeutic index in preclinical studies. NanoVation partners with leading pharmaceutical and biotech companies develop genetic medicines for previously untreatable diseases. NTx is delivering tomorrow's genetic medicines, today. For more information, please visit nanovationtx.com.

Nantes Université is a Higher Education institution that offers academic programs and conducts research.

Robert “Tex” Walker is a veterinary vaccines manufacturer that adheres to USDA guidelines.

Nephrogen is a therapeutics company developing transformative medicines for kidney disease. Their initial focus is on gene therapy for autosomal dominant polycystic kidney disease (ADPKD).

NeuExcell Therapeutics is a gene therapy company focusing on improving the lives of patients suffering from neurodegenerative diseases. We have discovered a disruptive technology that focuses on regenerating neurons in the brain. This powerful platform has the potential to transform patient outcomes and change lives. We are now rapidly expanding our R&D efforts, toward our goal of developing effective and safe treatments for serious neurodegenerative diseases including stroke, Huntington’s Disease, ALS, and Traumatic Brain Injury. Our experienced team is excited to dedicate themselves to contribute and bring innovative and effective treatments for those suffering from neurodegenerative conditions. It is our goal to improve the quality of life of millions of patients worldwide by using the power of gene therapy to restore damaged neuronal tissue.

At Neurophth, we walk from obstacle to obstacle without loss of enthusiasm. 在纽福斯公司，我们是从一个障碍走向另一个障碍而不丧失热情。 We are China’s first gene therapy company for ophthalmic diseases. Our mission is building a brighter future for patients by innovative gene therapies. Headquartered in Wuhan with subsidiaries in Shanghai, Suzhou, and USA, we are striving to discover and develop gene therapies for patients suffering from blindness and other eye diseases globally. We leverage our AAV and CMC platforms to regain vision in patients suffering from Leber’s hereditary optic neuropathy (LHON), autosomal dominant optic atrophy, vascular retinopathy (wAMD, DME, etc), optic neuropathy (glaucoma, etc) and other genetic diseases. Our most advanced investigational candidate, NR082 (rAAV2-ND4), in development for the treatment of LHON associated with ND4 mutation using a single administration through less invasive intravitreal injection, has received orphan disease designations in the U.S. We are always seeking highly motivated and talented individuals with diverse experiences, abilities and interests who are seeking to inspire the world and help patients to restore vision to join us on our journey. To learn more about Neurophth and our open positions, visit www.neurophth.com.

A global biotech company commited to developing new and effective therapies for psychiatric and neurological disorders affecting millions of patients in need. We design novel CNS-tropic AAV capsids and innovative gene expression cassettes to achieve efficient, effective, and safe gene therapies for severe CNS disorders.

Based in Lausanne, Switzerland, NewBiologix SA is a technology innovation company developing a proprietary and breakthrough DNA integration platform for the advanced engineering of human and mammalian cell lines for the improved production of viral vectors used in gene therapies.

Low-cost, rapid whole genome sequencing is the future of infection control and clinical microbiology. Next Gen Diagnostics offers the low cost, high throughput, and automated sequencing and bioinformatics for a range of application, including tracking the transmission of disease in hospitals and pathogen surveillance for public health labs.

Niba Labs is well known for quantitative assays development, targeting specific regions of any kinds of nucleic acid constructs (i.e. digital PCR assays for viral vector genome quantification). Additionally, we can also develop custom advanced multiplex approaches that can help investigate sequence integrity. Our mission is to advance analytical approaches for genome integrity evaluations.

Northeastern University is a private research university with its main campus in Boston, and multiple satellite campuses.

Novartis is reimagining medicine to improve and extend people’s lives. We deliver high-value medicines that alleviate society’s greatest disease burdens through technology leadership in R&D and novel access approaches. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. About 106,000 people of more than 140 nationalities work together to bring Novartis products to nearly 800 million people around the world.

Nuevocor is a new biotech start-up in Singapore striving to develop gene therapies for genetic cardiomyopathies. It will leverage a deep understanding of cardiac cell biology, as well as a technology platform encompassing novel gene therapy vectors and proteomics-based target discovery

NunaBio is a privately-owned specialist DNA synthesis and development company. We are engaged in CRO (Contract Research Organizations) and CDMO (Contract Development and Manufacturing Organisation) activities. The company has built a solid reputation for its R&D flexibility and innovative approach to complex and challenging projects. NunaBio's core expertise is research and development into cell-free DNA synthesis, process optimisation, route design, scale-up and small-scale manufacture for clinical studies.

Leverage nVector's advanced Adeno-Associated Virus platform to quick-launch your gene therapy strategy or accelerate existing initiatives. We enhance testing and trials with superior diagnostic biomarkers.

NysnoBio ia dedicated to developing the therapeutic potential of the Parkin protein for patients with serious medical conditions. We envision a future where the power of the Parkin pathway is directed toward specific disease areas, where the activation of the repair pathway can have maximum therapeutic benefits. Our core team of industry professionals and network of academic experts are working together to deliver on the scope of therapeutic potential for Parkin cellular repair.

Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one to many” and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the U.S. market.

Okogen is a clinical stage ophthalmic pharmaceutical company. Our lead product is in clinical trials as a “one stop shop” treatment for all forms of acute infectious conjunctivitis. Okogen is focused on developing ophthalmic therapeutics for unmet needs in ophthalmic infectious diseases.

Oncolys BioPharma Inc. is a Tokyo-based bioventure established in Japan in March 2004. Its mission is to contribute to the advancement of medical science in the world through bringing innovation to the existing treatments for cancer and serious infectious diseases, using its virology-based new drug discovery technologies. The company operates in two business segments. Pharmaceutical Business segment is engaged in the research, development, manufacture and marketing of novel drugs. Its major pipeline Telomelysin (OBP-301), an oncolytic adenovirus immunotherapy is in clinical development targeting various types of solid tumors including melanoma , HCC and esophageal cancer. Along with Telomelysin, an anti-HIV drug OBP-601 (Censavudine) and a novel HDAC inhibitor OBP-801 are also under development in the United States. Diagnostics Business segment is engaged in the research, development, manufacture and marketing of CTC (Circulating Tumor Cell) detection adenovirus TelomeScan, as well as the provision of inspection services. Oncolys was listed on the Tokyo Stock Exchange Mothers Market in December 2014 and has a clinical laboratory center in Kobe, Japan and a subsidiary in New Jersey, USA.

Oregon Health & Science University is dedicated to improving the health and quality of life for all Oregonians through excellence, innovation and leadership in health care, education and research.OHSU's singular focus on improving health starts with Oregonians and has global impact. They are the only academic health center in Oregon, but they are also nationally distinct as a university dedicated solely to advancing health sciences. This allows them to concentrate resources on research to prevent and cure disease, on education that prepares physicians, dentists, nurses and other health professionals to succeed in an evolving health care environment, and on patient care that incorporates the latest knowledge and discoveries.Based in Portland, they have 16,000 employees, operate the top-ranked adult and children's hospitals in Oregon, and secure competitive research funding of more than $400 million a year. As a public organization, they provide community outreach and services for Oregon's most vulnerable populations.

OriGene Technologies was founded as a research tool company focused on the creation of the largest commercial collection of full-length human cDNAs in a standard expression vector. The availability of the complete human genome sequence and the subsequent development of genome-based tools have enabled the identification of relevant drug targets through system biology approaches. OriGene's vision is to prepare comprehensive, genome-wide research tools and technology platforms to enable scientists to study complete biological pathways, thus enabling a better understanding of disease mechanisms including cancer and stem cell research. OriGene Technologies uses high-throughput, genome wide approach to develop products for pharmaceutical, biotechnology, and academic research. Our flagship product is the cDNA clone collection, a searchable gene bank of over 30,000 human full-length TrueClone cDNA collections and over 25,000 TrueORF cDNA clones. From our TrueORF cDNA clones, we have developed the largest offering of full-length human proteins expressed in mammalian cells, ideal for functional studies. In 2010, OriGene initiated the TrueMAB project to develop mouse monoclonal antibodies against protein antigens to develop protein assays for every human protein. In addition, OriGene offers unique gene expression products such as TissueScan cancer tissue qPCR arrays and tissue biorepository for biomarker discovery and validation OriGene is committed to its mission to be “Your Gene Company”, supplying everything a researcher needs for gene-based research. Visit us at https://www.origene.com

OXB (LSE: OXB) is a global quality and innovation-led contract development and manufacturing organisation (CDMO) in cell and gene therapy with a mission to enable its clients to deliver life changing therapies to patients around the world. One of the original pioneers in cell and gene therapy, OXB has 30 years of experience in viral vectors; the driving force behind the majority of cell and gene therapies. OXB collaborates with some of the world’s most innovative pharmaceutical and biotechnology companies, providing viral vector development and manufacturing expertise in lentivirus, adeno-associated virus (AAV), adenovirus and other viral vector types. OXB's world-class capabilities span from early-stage development to commercialisation. These capabilities are supported by robust quality-assurance systems, analytical methods and depth of regulatory expertise. OXB offers a vast number of unique technologies for viral vector manufacturing, including a 4th generation lentiviral vector system (the TetraVecta™ system), dual plasmid system for AAV production, suspension and perfusion process using process enhancers and stable producer and packaging cell lines.

Oxford Genetics is a synthetic biology company focused on developing novel technologies to overcome challenges associated with the discovery, development and production of biologics, gene therapies, cell therapies and vaccines.

At PacBio, we’re devoted to empowering you. Pioneering the future through biology takes vision. You have to look beyond the status quo to what’s possible. We know your vision can reveal the full potential of the genome, advance human health and the health of our planet. And that is why your vision and pursuit are at the very center of what our company does — our product design, our customer support model, our partnerships, and even our identity. We create the world’s most advanced sequencing systems to provide you the most complete and accurate view of genomes, transcriptomes, and epigenomes. Our founders invented a new way to study the synthesis and regulation of DNA, RNA, and proteins. Driven by a desire to advance human health, we’re harnessing advances in biochemistry, optics, nanofabrication, and more. We are passionate about developing products that empower scientists to explore the full spectrum of genetic variation in any organism — from unraveling the mystery of rare diseases to improving the world’s food supply. With world-class service and support, we keep your research progressing so you can focus on finding answers. We encourage you to see the whole picture — and consider us the partner you trust to see it too.

At PacBio, we’re devoted to empowering you. Pioneering the future through biology takes vision. You have to look beyond the status quo to what’s possible. We know your vision can reveal the full potential of the genome, advance human health and the health of our planet. And that is why your vision and pursuit are at the very center of what our company does — our product design, our customer support model, our partnerships, and even our identity. We create the world’s most advanced sequencing systems to provide you the most complete and accurate view of genomes, transcriptomes, and epigenomes. Our founders invented a new way to study the synthesis and regulation of DNA, RNA, and proteins. Driven by a desire to advance human health, we’re harnessing advances in biochemistry, optics, nanofabrication, and more. We are passionate about developing products that empower scientists to explore the full spectrum of genetic variation in any organism — from unraveling the mystery of rare diseases to improving the world’s food supply. With world-class service and support, we keep your research progressing so you can focus on finding answers. We encourage you to see the whole picture — and consider us the partner you trust to see it too.

Pacira BioSciences Inc. is a leading provider of non-opioid pain management and regenerative health solutions. We are dedicated to offering patients—and the clinicians who treat them—cutting-edge therapies to treat pain with the potential to reduce or eliminate the need for opioids. Founded in 2006 and headquartered in Tampa, FL, Pacira has offices in Parsippany, NJ as well as San Diego, CA. With more than 700 employees who share a passion for delivering improved patient care and reducing the need for opioids, Pacira is here to challenge and transform the way we think about – and treat – pain. We are committed to providing solutions that address unmet medical need and improve clinical results, and empowering patients and healthcare providers to choose non-opioid options. Our non-opioid product portfolio is aimed at opioid minimization, long-lasting pain control, and enhanced recovery. Find out why we aim for change at Pacira.com/commitments

PackGene is a world-leading AAV vector packaging CRO and CDMO. Founded in Massachusetts, USA, PackGene now has offices in Boston, Houston and Zurich. We work with customers to support gene therapy programs from early-stage R&D, preclinical development, to IND studies. Our mission is to accelerate gene therapy product development by providing an integrated one-stop solution including plasmid, viral vector, fill-finish and QC analytical services for the gene therapy industry.

Catalent Inc. (NYSE: CTLT), an S&P 500® company, is the global leader in enabling pharma, biotech, and consumer health partners to optimize product development, launch, and full life-cycle supply for patients around the world. With broad and deep scale and expertise in development sciences, delivery technologies, and multi-modality manufacturing, Catalent is a preferred industry partner for personalized medicines, consumer health brand extensions, and blockbuster drugs. Catalent supports our partners in introducing 200 new products and manufacturing over 70 billion doses each year. Since becoming a publicly traded company in 2014, we have grown to become a community of approximately 19,000+ workers who support over 7,000 products for over 1,000 customers around the world. We share a common goal: to put patients first and help people around the world live better and healthier lives. Catalyst + Talent. Our name combines these ideas. From drug and biologic development services to delivery technologies to supply solutions, we are the catalyst for your success. We have the deepest expertise, the broadest offerings, and the most innovative technologies to help you get more molecules to market faster, enhance product performance, and provide superior, reliable manufacturing and packaging results. more products. better treatments. reliably supplied.™ Your talents, ideas, and passion are essential to our mission: to develop, manufacture, and supply products that help people live better, healthier lives. Join us! Catalent hires people with a passion to make a difference to the health of millions of people globally. Your expertise, coupled with Catalent’s advanced technologies and collaboration with thousands of innovative pharmaceutical, biotech, and healthcare companies, will help bring life-enhancing products to life. Grow with us. Be challenged. Make a personal impact. Visit https://careers.catalent.com/us/en to explore career opportunities

Passage Bio is a genetic medicines company based in Philadelphia, PA that is developing a deep pipeline of adeno-associated virus (AAV) delivered therapeutics for the treatment of rare monogenic central nervous system disorders (CNS). We seek to deliver transformative therapies to patients by leveraging our team’s decades of experience, including our founders, as well as the transformative potential genetic medicine technology to develop treatments that improve outcomes for patients with serious, life-threatening, rare CNS disorders.

Paul-Ehrlich-Institut is a healthcare regulatory agency that oversees pharmaceuticals, approves vaccines, conducts biomedical research, and protects public health.

PepGen Inc., a clinical-stage biotechnology company, focuses on the development of oligonucleotide therapeutics for use in the treatment of severe neuromuscular and neurologic diseases. Its lead product candidate is PGN-EDO51, an EDO peptide that id in Phase I clinical trial to treat individuals with Duchenne muscular dystrophy (DMD). The company is also developing PGN-EDODM1, an EDO peptide-conjugated PMO, which is in Phase 1 clinical trial for the treatment of myotonic dystrophy type 1, as well as EDO therapeutic candidates, such as PGN-EDO53, PGN-EDO45, and PGN-EDO44 for the treatment of DMD. PepGen Inc. was founded in 2018 and is headquartered in Boston, Massachusetts.

Pfizer Inc. discovers, develops, manufactures, markets, distributes, and sells biopharmaceutical products worldwide. It offers medicines and vaccines in various therapeutic areas, including cardiovascular metabolic and women’s health under the Premarin family and Eliquis brands; biologics, small molecules, immunotherapies, and biosimilars under the Ibrance, Xtandi, Sutent, Inlyta, Retacrit, Lorbrena, and Braftovi brands; and sterile injectable and anti-infective medicines, and oral COVID-19 treatment under the Sulperazon, Medrol, Zavicefta, Zithromax, Vfend, Panzyga, and Paxlovid brands. The company also provides medicines and vaccines in various therapeutic areas, such as pneumococcal disease, meningococcal disease, tick-borne encephalitis, and COVID-19 under the Comirnaty/BNT162b2, Nimenrix, FSME/IMMUN-TicoVac, Trumenba, and the Prevnar family brands; biosimilars for chronic immune and inflammatory diseases under the Xeljanz, Enbrel, Inflectra, Eucrisa/Staquis, and Cibinqo brands; and amyloidosis, hemophilia, and endocrine diseases under the Vyndaqel/Vyndamax, BeneFIX, and Genotropin brands. In addition, the company is involved in the contract manufacturing business. It serves wholesalers, retailers, hospitals, clinics, government agencies, pharmacies, and individual provider offices, as well as disease control and prevention centers. The company has collaboration agreements with Bristol-Myers Squibb Company; Astellas Pharma US, Inc.; Myovant Sciences Ltd.; Akcea Therapeutics, Inc; Merck KGaA; Valneva SE; BioNTech SE; and Arvinas, Inc. Pfizer Inc. was founded in 1849 and is headquartered in New York, New York.

PhageNova Bio is a company that has developed a targeted gene delivery vector. The company is engaged in pioneering targeted gene expression for gene therapy.

PlasmidFactory GmbH, based in Bielefeld, Germany, is the leading contract manufacturer of plasmid and minicircle DNA. The company offers the production of plasmids and minicircles in several quality grades: Research Grade and ccc Grade for research purposes and pre-clinical applications, High Quality (HQ) Grade as starting material for e.g. GMP production of RNA and viral vectors and GMP Grade for clinical applications. PlasmidFactory has its own separate, dedicated HQ production area with new laboratories to produce highly purified plasmid DNA. To avoid cross-contamination, only one plasmid per time is produced there. Besides production of individual plasmids and minicircles, In Stock products that are deliverable immediately off the shelf, are also provided: e.g. reporter genes (gfp, lacZ, luc), AAV Helper & Packaging vectors (pDG/pDP family, several serotypes) or pEPI plasmids (containing S/MAR elements). PlasmidFactory is working on several research topics – some in close co-operation with industrial and academic partners.

We sequence whole plasmids, amplicons, whole bacterial genomes, and colonies overnight, without the need for primers and minipreps. Plasmidsaurus is on a mission to accelerate new cures and promote a healthier planet by unlocking new levels of productivity in scientists using DNA tools to bring their ideas to life. The company’s DNA sequencing services are used daily by thousands of innovators, including Nobel prize winners, dynamic biotech startups, pharmaceutical companies, research labs, and DIY biohackers. Plasmidsaurus’s global network of labs operates day and night to enable world-changing discoveries.

Politecnico Milano is a scientific-technological university which trains engineers, architects and designers. The University has always focused on the quality and innovation of its teaching and research, developing a fruitful relationship with business and productive world by means of experimental research and technological transfer. Research has always been linked to didactics and is a priority commitment which has allowed Politecnico Milano to achieve high quality results at an international level as to join the university to the business world. Research constitutes a parallel path to that formed by cooperation and alliances with the industrial system. Knowing the world in which you are going to work is a vital requirement for training students. By referring back to the needs of the industrial world and public administration, research is facilitated in following new paths and dealing with the need for constant and rapid innovation. The alliance with the industrial world, also favored by Fondazione Politecnico and by consortiums to which Politecnico belongs, allows the university to follow the vocation of the territories in which it operates and to be a stimulus for their development. The challenge which is being met today projects this tradition which is strongly rooted in the territory beyond the borders of the country, in a relationship which is developing first of all at the European level with the objective of contributing to the creation of a single professional training market. Politecnico takes part in several research, sites and training projects collaborating with the most qualified European universities. Politecnico's contribution is increasingly being extended to other countries: from North America to Southeast Asia to Eastern Europe. Today the drive to internationalization sees Politecnico take part in the European and world network of leading technical universities and offers several beside many which are entirely taught in English.

Polyplus, part of Sartorius, is a leading upstream solutions provider for advanced biologic and cell and gene therapy production from research to commercial scale. An innovator in nucleic acid delivery, the legacy portfolio features process-centric transfection reagents, kits, and support services for viral and non-viral delivery. Custom plasmid vector design and plasmid and protein manufacturing was integrated into the offer in 2022 to expand the products and services portfolio to help the industry optimize process economics while meeting strict scientific and regulatory standards.

Our mission at Poseida Therapeutics is to create cell and gene therapies with the capacity to cure. We are developing novel products using our broad gene engineering platform technologies, with the goal of bringing new and better treatments to patients with serious diseases, first in the blood-based cancer Multiple Myeloma and next in solid tumors like prostate cancer.

Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization.

Precision BioSciences, Inc. (Nasdaq: DTIL), is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform designed with therapeutic safety, delivery, and control in mind. Using ARCUS, Precision is developing allogeneic CAR T and in vivo gene correction therapies for cancer and infectious diseases.

Prevail Therapeutics was acquired by Eli Lilly and Company. At Prevail Therapeutics, we are developing a pipeline of gene therapies to slow or stop the underlying disease process for patients with Parkinson’s disease and other neurodegenerative disorders. We are leveraging recent breakthroughs in human genetics that point to potential disease-modifying targets for neurodegenerative diseases. Our programs utilize a precision medicine approach targeting patient populations with urgent unmet needs, where there are currently no available therapies that modify the progressive course of their disorders.

Prime Medicine was founded to bring the promise of gene editing to patients. We use Prime Editing, a next-generation technology that can “search and replace” to restore normal genetic function almost anywhere in the genome. Prime Editing searches to find the precise place in the genome to edit and replaces the segment of faulty DNA with a correct copy of DNA. A single Prime Editor can correct the individual mutations found across patients, meaning Prime Editing can potentially address more than 90 percent of known disease-causing genetic mutations. We envision a world where this technology can cure, halt, and ultimately prevent genetic diseases, providing lifelong benefit to patients. Prime Medicine offers the opportunity to shape the future of gene editing and transform how medicines are used to treat disease. Realizing the promise of Prime Editing requires a talented team with diversity of viewpoints and expertise. We’re building a group of dedicated, scientifically curious individuals who are passionate about helping patients live longer, healthier lives.

Priothera - We are dedicated to improving the lives of patients suffering from hematological malignancies by delivering an innovative immune modulator to enhance the curative potential of allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Our commitment to scientists is to support them in making new scientific discoveries and groundbreaking research within the life sciences and gene therapy. It is our underlying mission, to help progress new therapies safely, quickly and affordably, so together we can provide solutions to people suffering from poor health and affected by disease every day. PROGEN is made up of a team of experts within AAV and the life sciences and is partnered with gene therapy experts worldwide. We are more than just a manufacturer of antibodies, proteins and ELISAs kits. We strive to understand what scientists need, so we can create solutions and ultimately deliver high quality antibody and exclusive AAV products, which solve your research challenges within academia, biotech and pharma, and ensure PROGEN can continue to be a trusted and reliable partner. Visit our website www.progen.com Imprint: https://bit.ly/3uEZf2h Privacy Statement: https://bit.ly/3dMSgxm

ProtaGene is a world-leading CRO partner for the biopharmaceutical and gene and cell therapy industries. From research through product commercialization, we provide the most advanced, integrated, and complete protein and gene analytic capabilities and packages for biologic therapeutics as well as gene and cell therapy platforms. Our deep history of analytical leadership in protein-based therapeutics stems from the merger of Protagen Protein Services in Europe and BioAnalytix in the US. In 2021, GeneWerk’s unique capabilities in vector safety, integration site analysis, and bioinformatics were added to our portfolio, establishing ProtaGene. Our combined protein- and gene-based analytical platforms make it the leading analytic service provider across the biopharmaceutical and gene and cell therapy development spaces. The organization operates four sites in Europe and North America and works in advanced therapeutic platforms with leading biopharmaceutical and gene therapy companies worldwide.

ProteoNic is a biotechnology company active in development and licensing of DNA technology for cell line generation and recombinant protein production for pharmaceutical applications. We offer premium vector technology that substantially boosts production levels of therapeutic proteins and other biologics. Cost savings are immediate and sizeable, freeing up production capacity and enabling commercially viable production of difficult to express proteins. ProteoNic's 2G UNic™ drives higher production for all types of therapeutic protein products, including monoclonal antibodies, biosimilars and more difficult-to-express (DTE) proteins, such as Fc-fusion proteins and bi-specifics, contributing to their economic viability. Our patented technology does not require modifications to existing production systems or work flows and is compatible with all production cell lines and expression systems used in the industry. Independent technical and commercial validation by third parties, including top tier biopharma, demonstrates severalfold productivity level increases that translate into multi-million Euros in annual cost savings. The technology has wide ranging potential to boost production efficiencies in other important applications, including viral vector production, protein production in yeast and technologies which impact product quality. The company is headquartered at the BioScience Park in Leiden, the Netherlands and has a Boston, USA office for Business Development in the North American market.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines for the treatment of rare disorders. The company offers Translarna (ataluren) and Emflaza (deflazacort) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients. It is developing Translarna, which is in Phase 2 clinical study for the treatment of nonsense mutation aniridia and nonsense mutation Dravet syndrome/CDKL5; and RG7916 and RO7034067 for the treatment of patients with spinal muscular atrophy, as well as PTC596 and PTC299, a small molecule dihydrooratate dehydrogenase (DHODH) inhibitor that inhibits de novo pyrimidine nucleotide synthesis, which is in Phase 1 clinical development stage to treat cancer patients. The company is also developing gene therapy product candidate that include PTC-AADC for the treatment of Aromatic L-amino acid decarboxylase deficiency. It has collaborations with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., and the Spinal Muscular Atrophy Foundation to develop and commercialize compounds identified under its spinal muscular atrophy sponsored research program; research collaboration with Massachusetts General Hospital for the treatment of rare genetic disorders resulting from pre-mRNA; and Akcea Therapeutics, Inc. to commercialize Tegsedi and Waylivra for the treatment of rare diseases in Latin America and the Caribbean. PTC Therapeutics, Inc. was founded in 1998 and is headquartered in South Plainfield, New Jersey.

Purespring, which launched in November 2020 with £45 million of funding from Syncona Ltd, is the first company to be directly targeting the podocyte, a specialized kidney cell implicated in many kidney diseases, through AAV gene therapy. This approach is based on the work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol, where he heads a world leading group researching glomerular diseases.

Puresyn, Inc. is focused on and dedicated to providing manufacturing services capable of producing high quality DNA which can be used for many applications including GMP manufacture of gene therapy products.

PYC Therapeutics (ASX: PYC) is a clinical stage biotechnology company pioneering a new generation of RNA therapeutics for genetic diseases. It works by combining two complementary platforms: RNA-based drug design and PYC’s proprietary drug delivery technology. PYC's library of naturally derived cell penetrating peptides provide its delivery platform to overcome the major challenges of RNA therapeutics - getting into the target cell. PYC believes its PPMO (Peptide conjugated Phosphorodiamidate Morpholino Oligomer) technology enables a safer and more effective RNA therapeutic to address the underlying drivers of a range of genetic diseases for which no treatment solutions exist today. The Company is leveraging its leading-edge science to develop a pipeline of novel therapies including two programs focused on inherited eye diseases and preclinical discovery efforts focused on neurodegenerative diseases. PYC’s discovery and laboratory operations are located in Australia, and the Company’s preclinical and clinical operations are based in San Fransisco California.

Queen's University is a Higher Education institution that offers a wide range of academic programs and research opportunities.

Developing state-of-the-art AAV vectors for gene therapy

Rainbow Company Youth Theatre is a nationally acclaimed, award-winning youth theatre group funded by the city of Las Vegas. For more than 45 years, the Rainbow Company has been providing quality theatre experiences for young people and their families.

Ray Therapeutics Inc (RayTx) is a late-stage preclinical Bay Area biotechnology company pioneering the first-to-market precision optogenetic therapy for vision restoration that is highly active in ambient lighting (and will not require light-enhancing goggles). Our studies have shown for the first time that our proprietary ChRown optogenetic protein can restore high levels of visual acuity, contrast sensitivity and visual field. RayTx lead asset, RTx-015, is expected to address the huge unmet medical in people suffering from many forms of retinal blindness (independent of the underlying genetic cause), including those with advanced age-related macular degeneration. RayTx has partnered with a world-leading AAV gene therapy manufacturer is utilizing their proprietary commercial manufacturing process and analytics to maximally de-risk clinical development. RayTx anticipates clinical trials will begin by the end of 2023 in our initial indication, retinitis pigmentosa.

Regel’s technology utilizes a deactivated Cas system (dCas) which targets the epigenome without editing or damaging the DNA. This approach harnesses the natural mechanisms of gene regulation, allowing for efficient and permanent restoration of normal gene expression.

Regeneron Pharmaceuticals, Inc. discovers, invents, develops, manufactures, and commercializes medicines for treating various diseases worldwide. The company’s products include EYLEA injection to treat wet age-related macular degeneration and diabetic macular edema; myopic choroidal neovascularization; and diabetic retinopathy, as well as macular edema following retinal vein occlusion, including macular edema following central retinal vein occlusion and macular edema following branch retinal vein occlusion. It also provides Dupixent injection to treat atopic dermatitis and asthma in adults and pediatrics; Libtayo injection to treat metastatic or locally advanced cutaneous squamous cell carcinoma;Praluent injection for heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease in adults; REGEN-COV for covid-19; and Kevzara solution for treating rheumatoid arthritis in adults. In addition, the company offers Inmazeb injection for infection caused by Zaire ebolavirus; ARCALYST injection for cryopyrin-associated periodic syndromes, including familial cold auto-inflammatory syndrome and muckle-wells syndrome; and ZALTRAP injection for intravenous infusion to treat metastatic colorectal cancer; and develops product candidates for treating patients with eye, allergic and inflammatory, cardiovascular and metabolic, infectious, and rare diseases; and cancer, pain, and hematologic conditions. It has collaboration and license agreements with Sanofi; Bayer; Teva Pharmaceutical Industries Ltd.; Mitsubishi Tanabe Pharma Corporation; Alnylam Pharmaceuticals, Inc.; Roche Pharmaceuticals; and Kiniksa Pharmaceuticals, Ltd., as well as has an agreement with the U.S. Department of Health and Human Services, as well as with Zai Lab Limited; Intellia Therapeutics, Inc.; Biomedical Advanced Research Development Authority; and AstraZeneca PLC. The company was incorporated in 1988 and is headquartered in Tarrytown, New York.

REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno‐associated virus (AAV) gene therapy. REGENXBIO's NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO's mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO's NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third‐party NAV Technology Platform licensees.

ReiThera is a company devoted to developing and producing biopharmaceutical products based on gene delivery technologies doe advanced therapies, in order to prevent and treat several serious or life-threatening diseases.

Rejuvenate Bio is targeting the core drivers of chronic age-related diseases by unlocking the power of gene expression and epigenetic reprogramming to reverse pre-existing heart disease, metabolic disease, and kidney failure in humans.

Remedium Bio is a gene therapy company focused on the development of treatments for large unmet clinical needs. To enable this, we developed a revolutionary gene therapy platform technology, which enables safe, effective, and durable delivery of therapeutic genes while allowing for simple post-treatment dose adjustment. The Prometheus™ adjustable gene therapy platform is able to safely and durably replace multi-injection subcutaneously administered biologics, with a single treatment, at a fraction of the cost, and without genetic integration. Our lead product is a disease-modifying treatment for Osteoarthritis that is based on the only clinically validated chondro-regenerative mechanism. Remedium’s pipeline includes gene therapy treatments in the fields of Weight Loss, Type 2 Diabetes, and Stroke.

Founded by pioneers in biopharmaceuticals, Renova Therapeutics is developing a portfolio of definitive gene and peptide therapies to restore health to people suffering from cardiovascular and metabolic diseases. Our investigational therapies are based on carefully validated and proprietary research into disease mechanisms and the founders’ nearly 50 combined years of work in the gene therapy field. This experience enables us to generate an entirely original platform of therapeutics and delivery systems that in a single dose are designed to provide durable, life-long treatments for chronic diseases. Our scientific research, led by Dr. H. Kirk Hammond, appears in numerous peer-reviewed articles that establish the scientific rationale and evidence supporting the development of our pipeline and our novel approach to delivering gene therapies. The first indications the company is pursuing are gene therapy treatments for congestive heart failure (CHF) and type 2 diabetes, two of the most common and devastating chronic diseases in the world. The company’s lead product, RT-100, is a treatment that delivers a therapeutic gene directly to the heart during a routine outpatient procedure and has the potential to increase heart function in millions of patients with CHF. The Renova Therapeutics pipeline also includes a groundbreaking gene therapy in preclinical stage for sufferers of type 2 diabetes, as well as a peptide infusion therapy for the treatment of acute decompensated heart failure. OUR MISSION: Creating transformational gene and peptide therapies that treat the most prevalent diseases to restore health and renew life OUR VISION: A future in which we are able to permanently treat chronic diseases with gene and peptide therapies

Repair Biotechnologies is a preclinical biotechnology company focused on developing drugs for cholesterol and aging-related diseases. Our first-in-class Cholesterol Degrading Platform (CDP) technology is aimed at reversing atherosclerosis, familial hypercholesterolemias, and other conditions in which excess or modified cholesterol drives pathology.

Replay is a genome writing company, which aims to define the future of genomic medicine through reprogramming biology by writing and reprogramming biology.

Although ophthalmic medicine is progressing day by day, there are still some diseases neither treatment nor prevention methods has not been established yet. Ophthalmologists including me cannot do anything with such disease but just watching the patient go blind. As an ophthalmologist, I am struggling about the current limitation in ophthalmology and trying to overcome　this problem. There are more than 300,000 visually impaired patients in Japan, and the social cost is estimated to be more than 8 trillion yen, most of which are due to intractable diseases for which prevention nor treatment method has not yet been established. Please close your eyes. Can you imagine what the patients feel like losing their sight? It is said that vision occupies 80% of information that people obtain from the outside. Their quality of life will suffer considerably with blindness. In order to overcome these blindness diseases, we have established Restore Vision from Keio University School of Medicine to develop treatments for these intractable eye diseases using the latest technology. We will do our best for a bright future.

Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible. Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. Thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Roche has been recognised as the Group Leader in sustainability within the Pharmaceuticals, Biotechnology & Life Sciences Industry ten years in a row by the Dow Jones Sustainability Indices (DJSI). For more information, please visit https://careers.roche.com Read our community guidelines here: https://www.roche.com/some-guidelines.htm #Roche #Biotechnology #Pharmaceuticals #Diagnostics #Healthcare #PersonalisedHealthcare #GreatPlaceToWork #Innovation

Rocket Pharmaceuticals (NASDAQ: RCKT), Ltd is a biotechnology company focused on seeking cures through molecular and gene therapy. We are developing first-in-class treatments for patients with rare and often devastating orphan diseases using a lentiviral-based and adeno-associated virus (AAV) therapy approach. We have developed rewarding partnerships with a number of leading international research institutions as well as with centers of manufacturing and cell processing expertise, while building an internal team to drive the research, development, clinical and regulatory programs. Ultimately we aim to launch and globally commercialize innovative gene therapies in disease areas with high unmet need.

Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. We do this by building Vants – nimble, entrepreneurial biotech and healthcare technology companies with a unique approach to sourcing talent, aligning incentives, and deploying technology. The therapies in development across our family of companies target a wide range of diseases including uterine fibroids, endometriosis, prostate cancer, Parkinson’s disease, diabetes, pulmonary arterial hypertension, and multiple rare and fatal pediatric conditions. We focus on disease areas where the magnitude of R&D investment from industry is disproportionately low relative to societal medical needs. In addition to our biopharmaceutical subsidiaries, we also build technology-focused Vants focused on improving the process of developing and commercializing medicines.

Rznomics is developing safe and effective biopharmaceuticals in oncology, degenerative disease, and genetic disease based on RNA platform technology called trans-splicing ribozyme. Acknowledged by bio-focused institutions, Rznomics has raised \23.7 billion from series A& B and recently raised \37.2 billion from series C, resulting in a total of \60.9 billion of fundraising. Also, Rznomics received \8 billion for the new drug development research funding from the government. In 2022 1H, Rznomics’ RZ-001 IND submission for the HCC treatment was approved by MFDS, and the clinical trial for the RZ-001 is currently running. In addition to the RZ-001, Rznomics has competitive and innovative pipelines, such as glioblastoma multiforme, Alzheimer’s disease, genetic ocular disease (retinitis pigmentosa), and circular RNA technology. Rznomics is also actively partnering with global companies to discuss collaboration opportunities in co-research, co-development, and licensing.

Sangamo Therapeutics is a genomic medicine company conducting research & development across four distinct but complementary technology platforms – gene therapy, cell therapy, in vivo genome editing, and in vivo genome regulation. The optionality and diversity inherent to Sangamo’s technology platform enables us to design therapeutic approaches to resolve the underlying genetic causes of disease, using whichever technology is best suited to deliver that treatment. Because Sangamo’s technology platforms are related, we are applying learnings across studies to optimize each individual platform and uncover research and development paths forward. Sangamo is best known for developing our proprietary gene editing technology, zinc finger nucleases (ZFNs). Our technology team continues to optimize ZFNs for the three key dimensions of genome editing: precision, efficiency, and specificity. Ex vivo gene-edited cell therapy is the most straightforward application of ZFN technology. It builds on early experience with HIV clinical studies and is applied to hematological disorders and oncology. Our cell therapy portfolio also includes ex vivo engineered regulatory T-cells (CAR-Tregs) for application in autoimmunity and inflammation. The insights gained about molecular biology, cassette engineering, and delivery through in vivo application of ZFNs led to our gene therapy program using AAV, which is advancing in the clinic. Zinc finger protein engineering and AAV delivery are also foundational for our zinc finger protein-transcription factor (ZFP-TF) genome regulation technology, which we are applying in CNS diseases. With our robust history of genomic medicine firsts, unmatched technology platform, and growing manufacturing and clinical development capabilities, Sangamo believes we are the company to realize the vision of genomic medicine.

Sanofi, together with its subsidiaries, engages in the research, development, manufacture, and marketing of therapeutic solutions in the United States, Europe, and internationally. It operates through three segments: Pharmaceuticals, Vaccines, and Consumer Healthcare. The company provides specialty care products, including human monoclonal antibodies; products for multiple sclerosis, neurology, other inflammatory diseases, immunology, rare diseases, oncology, and rare blood disorders; medicines for diabetes; and cardiovascular and established prescription products. It also supplies poliomyelitis, pertussis, and hib pediatric vaccines; and influenza, adult booster, meningitis, and travel and endemic vaccines. In addition, the company offers allergy, cough and cold, pain, liver care, physical and mental wellness, probiotics, digestive, and nutritional products; and other products, such as daily body lotions, anti-itch products, moisturizing and soothing lotions, and body and foot creams, as well as powders for eczema. Further, it has various pharmaceutical products and vaccines in development stage. Sanofi has collaboration agreement with GlaxoSmithKline to develop a recombinant Covid-19 vaccine; and a research collaboration with Stanford University School of Medicine to advance the understanding of immunology and inflammation through open scientific exchange. It also has a collaboration and license option agreement with Prellis Biologics, Inc. The company was formerly known as Sanofi-Aventis and changed its name to Sanofi in May 2011. Sanofi was founded in 1973 and is headquartered in Paris, France.

Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For information on our Community Guidelines, please visit sarepta.com/community-guidelines. We want to share a reminder with all job seekers and candidates regarding the persistence of recruiting fraud. Please read a message about recruiting fraud and steps you can take to protect yourself here: https://www.sarepta.com/recruiting-fraud

We develop and manufactures market-leading CIM® monolithic chromatographic columns for the purification and analysis of large biomolecules, such as viruses, pDNA, and mRNA, which are applied in vaccines and gene therapies. Our Cornerstone® Biomanufacturing Development Services offer a comprehensive approach of integrated process development solutions and technology designed to improve the robustness and yield of large biomolecules production while improving the safety of therapeutic products.

Scout’s mission is to harness the genetic revolution transforming human medicine to deliver the future of veterinary medicine. By combining world-leading talent in animal health and gene therapy technology, we have built a development platform for one-time therapies that address major areas of unmet medical need in pet medicine. We are a global company supported by leading life sciences investors and an R&D collaboration with the renowned Gene Therapy Program at the University of Pennsylvania. About Our Currently Enrolling Study for Cats Scout’s first therapy in development focuses on the anemia associated with chronic kidney disease (CKD) in cats. Our study is currently enrolling cats at participating clinics across the U.S. It involves a single injection (followed by 70 days of monitoring) and may relieve a cat’s anemia for life. Learn more about our study and find a participating clinic: www.scoutbio.co/catEPOstudy.

Scribe is building and applying a suite of CRISPR technologies designed for therapeutic use into a single continually evolving and expanding genetic modification platform. Specifically, the company is working on NK cell therapies.

Sensorion is a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders Its clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) progressing in a planned Phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) and, with partner Cochlear Limited, a study of SENS-401 in patients scheduled for cochlear implantation. Sensorion has entered into a broad strategic collaboration with Institut Pasteur focused on the genetics of hearing. It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness including OTOF-GT, targeting deafness caused by a mutation of the gene encoding for otoferlin, and hearing loss related to mutation in GJB2 gene to potentially address important hearing loss segments in adults and children (GJB2-GT).

Shanghai Sunway Biotech is a biopharmaceutical company that develops, manufactures, and commercializes cancer biotherapeutics based on its oncolytic virus platform. The company has been involved in clinical trials and has received approval for oncolytic virus therapy for cancer treatment in China. Shanghai Sunway Biotech aims to provide innovative and effective treatments for cancer patients.

Shape Therapeutics is a biotechnology company that combines breakthroughs in AI, RNA technology, and synthetic biology to create safe, effective, and accessible medicines to help as many patients as possible. The company was founded with the mission to make programmable RNA medicines available for everyone. Shape Therapeutics is pioneering the field of programmable RNA Medicines.

Siren Biotechnology is combining two transformative therapeutic technologies – AAV gene therapy and cytokine immunotherapy - into a single, reimagined modality that overcomes key challenges and redefines how we destroy tumor cells and elicit anti-tumor immunity.

Headquartered in Rancho Cordova, Calif., SK pharmteco is a global contract development and manufacturing organization specializing in the production of active pharmaceutical ingredients (APIs), advanced intermediates, and cell and gene therapy for the pharmaceutical industry. SK pharmteco is comprised of six companies: SK Biotek Korea, SK Biotek Ireland, AMPAC Fine Chemicals, AMPAC Analytical Laboratories in the U.S., Yposkesi and the Center for Breakthrough Medicines.

Skyline Therapeutics is a biotechnology company that focuses on the discovery, development and delivery of innovative gene therapy treatments.

SOLA Biosciences (SOLA) offers a new proprietary, TapBoost® technology, to improve target protein production in various pharmaceutical companies and researchers that share our ultimate goal to deliver new therapeutics faster and more efficiently, at a lower overall cost to patients around the world. TapBoost technology is able to efficiently increase the production of target proteins such as antibodies and Fc-fusion proteins as well as many “difficult-to-express” recombinant proteins including therapeutic biologics. In addition to protein production, we anticipate additional pharmaceutical applications of TapBoost including new gene therapies for conformational diseases, drug screening with new disease target proteins, development of innovative biologics, and GMP manufacturing of commercial biologics. SOLA provides access to TapBoost to help promote pharmaceutical research and development in two ways: 1. Manufacture and provide needed proteins to researchers and pharmaceutical companies 2. Licensing Researchers and pharmaceutical companies can license TapBoost to produce needed proteins in-house. Please see our License page for more information.

Solid Biosciences (Solid) is a life science company singularly focused on solving Duchenne muscular dystrophy (Duchenne). We aim to be the center of excellence across the entire disease spectrum, serving as a catalyst to bring together those with the expertise in science, technology, disease management and care. Disease-focused and founded by a family directly impacted, our mandate is simple yet comprehensive - attack the roots of the disease and improve daily life for patients.

SonoThera is the world's first ultrasound-guided gene delivery platform designed to overcome limitations of current gene therapy approaches.

South East Technological University is a higher education institution in the South East region that focuses on technology, research, and innovation.

SparingVision is a genomic medicines company, translating pioneering science into vision-saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders at the Paris Vision Institute, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is a pipeline of gene independent treatments for rod-cone dystrophies. Lead products, SPVN06 and SPVN20, address mid and late stages of retinitis pigmentosa (RP)respectively. RP is the most common inherited retinal disease affecting two million people worldwide. These novel medicines could form the basis of a suite of new sight-saving treatments with potential applications across many other retinal diseases, regardless of genetic cause. The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised €60 million to date and its investors include 4BIO Capital, Advent France Biotechnology, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit www.sparingvision.com.

At Spark® Therapeutics, we believe in a world where no life is limited by disease.   We were born of innovation, springing from the curiosity, imagination and dedication of remarkable scientists and healthcare visionaries. Our mission is to unlock the power of gene therapy to accelerate healthcare transformation.     Since our founding, we have been committed to bringing a wide range of expertise to build a fully integrated gene therapy company focused on inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver, such as hemophilia. We are seeking talented individuals with diverse experiences, abilities and interests who have the curiosity, courage and drive to reimagine a new health care paradigm.     Join us on a journey through uncharted territory – seeking to bring gene therapy to people worldwide. The resilience of the people we serve is our inspiration to break barriers, as we strive to turn genes into medicine. Spark® is a member of the Roche Group.     Know that working at Spark Therapeutics is not just another job; it is a once-in-a-lifetime opportunity. We embrace the challenges before us and the uncertainty inherent in them. Ultimately, we are working to create a world where no life is limited by disease. To learn more about Spark and our open positions, visit www.sparktx.com. You can also find us on Twitter at @Spark_tx.  Spark’s social media sites are monitored from Monday to Friday during normal business hours. Questions or concerns should be directed here: https://sparktx.com/contact-us/.

Acquired by Sumitomo Dainippon Pharma of Japan. Spirovant Sciences is a gene therapy company focused on changing the course of cystic fibrosis and other respiratory diseases. The company's current focus is on developing novel gene therapies for cystic fibrosis and other pulmonary diseases. Spirovant is located in Philadelphia, PA's thriving Life Sciences Corridor and has a diverse management team, researchers, and partners.

SpliceBio is a biotechnology company exploiting Protein Splicing to develop the next generation of gene therapies. Our proprietary platform is based on technology developed in the Muir Lab at Princeton University after more than 20 years of pioneering intein and protein engineering research by our co-founders. The company is backed by UCB Ventures, Ysios Capital, NEA, Gilde Healthcare, Novartis Venture Fund and Asabys Partners.

We are a clinical stage oncology focused company developing novel gene therapies focused on dermatological and visceral cancers.

Stanford University is a Higher Education institution that focuses on research, innovation, and providing educational services.

St. Jude Children's Research Hospital is a healthcare institution specializing in pediatric medical research with a focus on cancer treatment.

Sudhin Biopharma Co. is a biotechnology company developing efficient continuous biological manufacturing processes for therapeutic biologicals, sweet proteins, cell therapies and biological roducts.

Syd Labs, a recombinant company based in Boston, Massachusetts, is dedicated to providing quality products and services for research and development in academy, industry, and government. We have always tried our best to let you, our valuable customers and partners, save money and work better. Syd Labs, Inc provides very comprehensive and competitive services for antibody services, from peptide synthesis, gene synthesis, protein expression, rabbit and mouse polyclonal antibody development, mouse, rat, and rabbit monoclonal antibody development, cloning variable regions of hybridoma cell lines, antibody humanization and engineering, stable cell line generation, ELISA assay development, and so on. The lowest prices are guaranteed. If you find any US company providing the same quality services with lower prices, we will return the difference to you.

At SBI, our mission is to accelerate your science by turning the latest insights and technologies into robust research products and services to advance exosome studies, biomarker research, gene delivery, SARS-CoV-2 studies, and more.

Takara Bio Inc. is committed to improving the human condition through biotechnology. Starting as the biomedical business of Takara Shuzo, a fermentation and beverage company founded in 1925 (now Takara Holdings Company, Inc.), Takara Bio has focused on developing research tools for the life sciences, cell and gene therapy technologies, and nutraceuticals since its launch in 1967. The three business units of TAKARA BIO INC are: • Genetic Engineering Research - manufactures and sells research reagents and scientific instruments used by biotechnology researchers worldwide, as well as contract research services. • Agribio - produces and sells health food products whose functionality have been proven by biotechnology, and conducts a mushroom business based on technologies for large-scale mushroom production. • Gene Medicine - conducts clinical development projects to work toward commercializing cell and gene therapies centered on a highly efficient gene transduction method and a lymphocyte expansion-culture system, both using RetroNectin® reagent.

Taysha Gene Therapies, Inc., a gene therapy company, focuses on developing and commercializing adeno-associated virus-based gene therapies for the treatment of monogenic diseases of the central nervous system. It primarily develops TSHA-101 for the treatment of GM2 gangliosidosis; TSHA-118 for the treatment of CLN1 disease; and TSHA-102 for the treatment of Rett syndrome. Taysha Gene Therapies, Inc. has a strategic partnership with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments; and collaboration with Yale University to advance mini-gene payloads for an AAV gene therapy for the treatment of neurodevelopmental disorders. The company was incorporated in 2019 and is based in Dallas, Texas.

TEAMEDON INTERNATIONAL, LLC is a company based out of 10134 TREBLE CT, ROCKVILLE, Maryland, United States.

Teknova makes solutions possible. Since 1996, Teknova has been innovating the manufacture of critical reagents for the life sciences industry to accelerate the discovery and development of novel therapies that will help people live longer, healthier lives. We offer fully customizable solutions for every stage of the workflow, supporting industry leaders in cell and gene therapy, molecular diagnostics, and synthetic biology. Our fast turnaround of high-quality agar plates, microbial culture media, buffers and reagents, and water helps our customers scale seamlessly from RUO to GMP. Headquartered in Hollister, California, with over 200,000 square feet of state-of-the-art facilities, Teknova's modular manufacturing platform was designed by our team of scientists, engineers, and quality control experts to efficiently produce the foundational ingredients for the discovery and commercialization of novel therapies.

Temple University is a higher education institution that offers a wide range of academic programs and degrees.

Tenaya Therapeutics, Inc. develops therapeutics for heart failure. It offers cellular regeneration platform, that enables in vivo reprogramming of cardiac fibroblasts into cardio myocytes by delivering proprietary transcription factors; gene therapy platform, that enables the targeted delivery of a payload to cardiac fibroblasts; and precision medicine platform, which provides personalized medicine approach to treat heart disease. The company was incorporated in 2016 and is based in South San Francisco, California.

Tenpoint Therapeutics Limited is a global, clinical-stage biotech company developing groundbreaking treatments to rejuvenate vision in the aging eye. Its pipeline includes paradigm-shifting treatments for ophthalmic indications with the greatest need and global market potential, including presbyopia, cataracts and geographic atrophy. Its lead asset, BRIMOCHOL PF, is a novel, pupil-modulating therapeutic designed to correct the loss of near vision associated with presbyopia, a condition that afflicts approximately 2 billion people globally. BRIMOCHOL PF has completed its first Phase 3 pivotal trial (BRIO-I) and will complete the second pivotal study, BRIO-II, in 2025. This topical ophthalmic is poised for launch in 2026. Tenpoint’s leadership team includes ophthalmic industry luminaries with track records of successful approvals and commercialization of blockbuster drugs. A privately held company, Tenpoint Therapeutics is backed by AdBio Partners, British Patient Capital, Eight Roads, EQT Life Sciences, F-Prime Capital, Hillhouse Capital Management, Qiming Venture Partners USA, Sofinnova Partners, and UCL Technology Fund.

Tessera Therapeutics is pioneering Gene Writing™—a new genome engineering technology that writes therapeutic messages into the genome to treat diseases at their source.

Texas Tech University Health Sciences Center is a higher education institution that offers comprehensive health sciences education and training, focusing on research and clinical practice to improve community health.

Theragene Pharmaceuticals is creating life-changing cancer and cardiology treatments through the development of our novel immunotherapy and Adeno-Associated Virus (AAV) gene therapy platforms. As a company operating in both the US and Asia, we deliver unique product development models, utilizing the strengths of each region, while delivering safe, effective treatments. Our mission is to advance cutting-edge science through efficient development plans while pursuing licensing or co-development collaborations.

Theriva Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat diseases in areas of high unmet need. The Company advanced into oncology through the acquisition of VCN Biosciences, S.L. (VCN), who have developed a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient's immune system. The Company's lead candidates are: (1) VCN-01, an oncolytic adenovirus designed to replicate selectively and aggressively within tumor cells, and to degrade the tumor stroma barrier that serves as a significant physical and immunosuppressive barrier to cancer treatment; (2) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent (a) microbiome damage, (b) Clostridioides difficile infection (CDI), (c) overgrowth of pathogenic organisms, (d) the emergence of antimicrobial resistance (AMR), and (e) acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients, and (3) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases.

Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue of approximately $40 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, increasing productivity in their laboratories, improving patient health through diagnostics or the development and manufacture of life-changing therapies, we are here to support them. Our global team delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services, Patheon and PPD.

TILT Biotherapeutics Ltd is a clinical stage company working in oncology towards enabling T-cell therapies and immune checkpoint inhibitors with oncolytic viruses. Embodiments of the technology will be used to enhance tumor infiltrating lymphocytes (TILs), chimeric antigen receptor T cells (CAR T) and immune checkpoint inhibiting antibody therapies. TILT was founded by Pr. Akseli Hemminki, who has published hundreds of papers in the field and treated almost 300 patients with 10 different oncolytic viruses. In 2013, Pr. Hemminki started his second company, TILT Biotherapeutics Ltd, with a technology based on his past findings in the lab, but more importantly in the clinic. The research conducted by TILT alone or in collaboration with some of world’s leading research groups with synergic technologies has generated very excellent results. The lead candidate is a clinical stage TNFα/IL2 armed oncolytic adenovirus (TILT-123 or Ad5/3-E2F-delta24-hTNFα-IRES-hIL2). Multiple Phase I clinical trials are ongoing. For contact: BD (at) tiltbio.com

Manufacturing.

Trogenix unites cutting-edge technologies in genomics, oncology, immunotherapy, and gene therapy to create a revolutionary therapeutic approach through its Odysseus platform. Using proven AAV vectors, our proprietary Synthetic Super-Enhancers (SSEs) are delivered directly to tumour cells without detection. Our SSEs enable unprecedented precision in gene control, effectively revealing cancer to the body's immune system and killing tumour cells. For any cells escaping Trogenix's technology, our 'Trojan horse' reawakens the immune system, countering recurrence and offering potentially curative 'one-and-done' treatments for aggressive tumours. With our lead asset showing curative potential in preclinical glioblastoma studies, we're poised to transform treatment paradigms across multiple cancer types and explore applications beyond oncology, such as regenerative medicine.

The vast majority of human disease is governed by the epigenome, the subtle machinery that controls the volume of gene expression in cells. Tune Therapeutics is pioneering the creation of epi-therapeutic medicines with its powerful and precise epigenomic control platform, TEMPO. Unlike genome editing, TEMPO dials the volume of gene expression up or down towards healthy levels, without breaking or permanently rewriting DNA – and it can do this in multiple genes at once. This genetic tuning approach allows us to change cell fate and function at will, unlocking the ability to reverse pathways of cancer, genetic disease and aging. Our approach effectively expands the potential reach of genetic medicine – from targeting a limited range of rare conditions, to overcoming thousands of complex and chronic diseases that currently have no curative treatment.

UCB – Inspired by patients. Driven by science. At UCB, we put our heart, soul and skills into making a difference for people living with severe diseases. We look to transform patients’ lives by pushing the boundaries of what is possible. With us, our talents makes the most of who they are, unlocking innovation and setting new standards for patients. Everything we do starts with a simple question: “How will this make a difference to the lives of people living with severe diseases?” And to find the answers, we connect globally with patients and their families living with the physical and social burdens of severe disease. These connections give us new perspectives, drive our innovation, and offer a hope for a new generation of therapies that will help to transform lives. UCB – The Facts With approximately 9000 employees in around 40 countries, we are a global biopharmaceutical company headquartered in Brussels, Belgium which invests more than 25% of revenue in cutting-edge scientific research to meet unmet patient needs. A few other reasons that make UCB an amazing place to work: - Promising pipeline that includes several novel molecules; - 2023 Key Financials: € 5.3 billion global revenue Specialties: Neurology, Immunology

Leading with purpose. Every day. “Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.” Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President We have an inclusive culture of value and respect. Ultragenyx brings novel products to those living with rare and ultrarare diseases with a focus on debilitating genetic diseases. Founded in 2010, we have rapidly built a diverse portfolio of approved therapies and products aimed at diseases with a high unmet medical need for which many have no approved therapies. It takes courage, care, talent, and dedication to make a meaningful impact for those living with rare diseases, their families and the rare disease community. We put our people first, so they can take care of the rare disease community. We purposefully cultivate and nurture a dynamic, supportive work environment where we encourage every team member to share their new ideas and help us unlock more possibilities. Our team is made up of exceptional and diverse people with a strong passion and commitment to helping those living with rare diseases. By embracing generosity, curiosity, inclusion, and humility we are constantly learning together—fostering an environment that supports profound growth and fulfillment. If you want to have a meaningful impact, do the best work of your career while having fun and growing professionally and personally, come join our team! Visit our community guidelines at: https://ultragenyx.co/community

uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with severe genetic diseases.

Valo Therapeutics (ValoTx) is a developer of novel immunotherapies in cancer and infectious disease. Our unique approach to immunotherapy, based on oncolytic viruses and viral vectors combined with disease-specific peptides, delivers highly adaptable and immunogenic therapeutic vaccination platforms. A spin-out company from the University of Helsinki, Finland, Valo Tx has assembled a talented team of immunotherapy experts, who together with the founding scientists have the necessary expertise to take its patented technology through clinical development and make it available to patients.

Vector Biolabs, a Fortis Life Sciences Company, is focused on developing robust gene delivery products through use of proprietary tools and technologies. Vector has been the leading CMO in research, discovery, and preclinical viral vector production for over 20 years, and has constructed and produced more than thirty thousand AAVs and adenovirus capsids.

VectorBuilder offer gene delivery technologies. VectorBuilder offers a full spectrum of gene delivery solutions covering virtually all research and clinical needs from bench to bedside

VectorY combines the therapeutic potential of antibodies and gene therapy to develop long-lasting therapeutic solutions for large disease areas of high unmet medical need. By combining state-of-the-art technologies and novel and scientific concepts, VectorY’s mission is to bring innovative therapies to patients worldwide. Founded in August 2020, and based in the Amsterdam Science Park, VectorY is a fully integrated gene therapy company focused on the development of innovative therapeutics such as vectorized antibodies for both CNS and somatic disorders, with a special focus on muscle diseases. With R&D and manufacturing facilities in Amsterdam, VectorY develops proprietary & partnered programs based on its novel AAV-based vectorized antibody & gene therapy platform. Product candidates are based on new vector technologies, which will enable the next generation of highly scalable manufacturing processes within VectorY’s own manufacturing facilities. Our manufacturing capabilities will include a state-of-the-art multi-product GMP facility in the Netherlands, with the capability to deliver suspension based AAV viral vector manufacturing of up to 2000L for both clinical and commercial supply.

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. We discovered and developed the first medicines to treat the underlying cause of cystic fibrosis (CF), a rare, life-threatening genetic disease. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases. Founded in 1989 in Cambridge, Massachusetts, our corporate headquarters is now located in Boston’s Innovation District, and our international headquarters is in London, United Kingdom. We currently employ approximately 3,500 people in the United States, Europe, Canada, Australia and Latin America with nearly two-thirds of our staff dedicated to research and development. Vertex is consistently recognized as one of the industry’s top places to work by Science Magazine, The Boston Globe, Boston Business Journal and the San Diego Business Journal. Our research and medicines have also received esteemed recognitions, including the Robert J. Beall Therapeutics Development Award, the French Prix Galien and the British Pharmacological Society awards.

Via Nova Therapeutics is committed to discovering novel therapies for viral diseases, especially those arising from acute & subacute infections.

ViGeneron was founded in 2017 as a spin-off of the Ludwig-Maximilians-University (LMU) Munich. At ViGeneron we are dedicated to develop innovative gene therapies based on our two unique and proprietary technology platforms. ViGeneron will leverage its two technology platforms to address unmet medical needs with in-house gene therapy programs for selected retinal disorders.

VintaBio’s development and manufacturing team has over a century of experience in the cell and gene therapy space and its members have played key roles in the development of the most advanced therapies on the market today. Located in the historic Navy Yards of Philadelphia – the epicenter of cell and gene therapy innovation – VintaBio specializes in developing and manufacturing consistent, high-quality viral vectors, a vital and currently underserved area of CGT development. The company was founded by Junwei Sun and Dr. Shangzhen Zhou, who led development of the AAV vectors behind the first two FDA-approved gene therapies, as well as the lentiviral vector for the first FDA-approved cell therapy. VintaBio has raised $64 million in venture funding, which has been used to develop a brand new 22,500 square foot facility in Philadelphia created specifically to facilitate efficient cell and gene therapy manufacturing.

Viralgen is a CDMOfor AAV production that supportslarge-scale manufacturing, fill-finish, and quality control in a GMP-certified environment

At Virion Therapeutics, We Believe a Future Free of Cancer and Chronic Infectious Diseases Is Within Reach​ Driven by science, our mission is to revolutionize the immunotherapy treatment landscape, focusing on the development of novel, adaptable, and accessible CD8+ T cell-based technologies.​ Our transformative platform is first in its class, offering a unique methodology that combines innovative and established approaches to elicit a superior CD8+ T cell response. Novel genetically encoded checkpoint modifiers are delivered alongside optimized target-specific antigens via an immunogenic and adaptable viral vector platform that promotes potent, prolonged, and broad CD8+ T cell responses. With potential applications across a range of cancers and chronic infectious diseases, our proprietary technology provides us with a robust pipeline. Our lead product, VRON-0200, is under investigation as a potential functional cure for the treatment of chronic hepatitis B virus (HBV) – a first-in-human clinical study is planned to initiate in late 2022. Founded in 2018 to advance technology licensed from The Wistar Institute, we have assembled a world-class management team and an industry-leading global Board of Directors, with proven experience in the biotechnology sector, as well as extensive preclinical and clinical expertise in antiviral, vaccine, oncology, and immuno-oncology.​

Vironexis is focused on transforming the future of cancer treatment by pioneering AAV-delivered T-cell immunotherapy. Our TransJoin™ AAV Gene Therapy Platform enables the creation of off-the-shelf, single-dose gene therapies designed to overcome the key challenges and shortcomings of current immunotherapies, including CAR-T and bispecific antibodies. Our current pipeline includes more than ten product candidates for blood-based cancers, solid tumor metastasis prevention, and a cancer vaccine. Our lead program, VNX-101, for the treatment of CD19+ acute lymphoblastic leukemia, is anticipated to begin clinical trials in the fourth quarter of 2024.

Visgenx is developing therapeutics based on increasing expression of a gene known as ELOVL2 which has been shown to regulate aging in the retina and other tissues. Our lead indication is Dry Age-Related Macular Degeneration (Dry AMD).

Vitalgen is a biopharmaceutical company that focuses on the research and development of gene therapeutics.

Vivet Therapeutics is dedicated to developing innovative gene therapy treatments for orphan diseases. Vivet Therapeutics is focused on optimizing gene therapy through collaborating with the Fundacion para la Investigacion Medica Aplicada (CIMA, Universidad de Navarra) to develop new AAV vectors specifically targeting the liver and generating new technologies to optimize gene delivery and long term expression.

Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more information, visit www.voyagertherapeutics.com. Voyager Therapeutics® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc.

Vybion developed a novel vectorized intrabody (INT41) for Huntington’s disease, a devastating inherited orphan disease that results in the progressive loss of both motor and cognitive function. INT41 is effective in both cell-based system and animal models. INT41 has Orphan Disease Designation from the FDA and a pre-IND meeting defined the basic elements of the IND enabling package. INT41 binds to an epitope that is unavailable in the functional molecule, but appears in the toxic degradation fragment that accumulates in the nucleus, binds to DNA via the polyglutamine and alters gene expression detectable years before the onset of symptoms. INT41 stabilizes this fragment and facilitates its degradation. In addition to Huntington’s disease, Vybion is testing INT41 as a therapy for spinobulbar muscular atrophy (SBMA) and spinocerebellar Ataxia 1, 3 and 7 (SCA), two closely related Orphan diseases with an identical target sequence and polyglutamine expansion as seen in Huntington’s disease.

Washington State University is an academic institution that offers higher education and research services.

Welgen Inc is a Biotechnology company located in 25 Winthrop St, Worcester, Massachusetts, United States.

Westlake Genetech (Hangzhou) Co., Ltd. is an innovative biotech company, a start-up from scientific research achievements of Westlake University. We focus on the rapid development of first-in-class drugs by integrating the 3H (high throughput, high precision, high dimension) biotechnological data production and deep learning modeling. By building technology-driven gene therapy technology platforms and developing differentiated self-developed gene therapy products, we are committed to mapping the full-process implementation of AI-powered gene therapy. Westlake Genetech adheres to the development strategy of "platform + pipeline" dual-wheel drive, actively invests in R&D innovation that combines technological leadership and industrial feasibility, and promotes the technological and commercial landing of AI technology in the vertical field of biomedicine. Our self-developed AI-powered full-chain gene therapy technology platform has evolved to generate a large number of non-liver-targeted AAV capsids, developed gene editing tools with independent intellectual property rights, explored new targets, and developed safe, efficient, and accessible gene therapy innovative therapies for rare diseases, tumors, and other diseases. With the platform's empowerment, it will effectively expand the scope of gene therapy indications, significantly shorten the pipeline development time, improve the pipeline R&D efficiency, and achieve the optimization of cost reduction and efficiency improvement and technology tool selection. We are rapidly advancing our technological achievements to the field of disease treatment, laying out ex vivo and in vivo gene therapy, developing differentiated gene therapy products, and solving more unmet clinical needs.

Founded in 2019, backed by Y-Combinator, WhiteLab Genomics stands at the convergence of computer sciences and biology, pioneering the accelerated development of genomic medicines. By leveraging their proprietary technology, WhiteLab Genomics analyzes complex biological data powered by AI to significantly reduce development timelines and mitigate associated risks. Based on exhaustive datasets, the platform provides in-silico simulations to discover, and design optimized payloads and vectors. WhiteLab aims to expedite the drug development process, cut costs, and accelerate the delivery of life-saving therapies to the market. The company collaborates with leading pharmaceutical companies such as Sanofi, academic institutions, and innovative biotechnology companies. Recognized for their contribution to advancing the field of gene and cell therapy, WhiteLab Genomics is part of the prestigious French government supported by French Tech 2030 program and recently joined the Bayer Co.Lab in Cambridge, MA, and Ginkgo Bioworks' Technology Network, marking major milestones in our international expansion.

WuXi Advanced Therapies - Accelerating Progress and Time to Market WuXi Advanced Therapies is a cell and gene therapy Contract Testing, Development and Manufacturing Organization (CTDMO) that is reducing the complexities of manufacturing by providing integrated platforms that enable cell and gene therapies to be developed, manufactured, and released faster and with greater predictability. Our Story By leveraging platforms and integrated testing our team provides expedited development and commercialization of cell, gene, protein and viral-based therapies. This approach enables new biopharmaceuticals to be developed, manufactured and released faster and with greater predictability, thereby reducing the complexities of high-touch, multi-vendor production models. Our solutions help clients overcome challenges to commercialization, including process development, manufacturing capacity, analytical development, and raw materials management. Multiple, scale-able, enabling platforms integrate manufacturing, process development and testing capabilities to provide greater predictability and speed to clinic. We support advanced therapy programs with extensive infrastructure and 400,000+ square feet of state-of-the-art, GMP-compliant facilities on our modern campus located at the Navy Yard in Philadelphia, Pennsylvania.

Xell is a Germany-based biotech company that develops, produces and markets media and feed supplements for gene therapeutics and vaccines.

Xencor, Inc., a clinical stage biopharmaceutical company, focuses on the discovery and development of engineered monoclonal antibody and other protein therapeutics to treat severe and life threatening diseases. It develops its antibody product candidates to treat cancer and autoimmune diseases. The company’s product candidates include XmAb5871, an immune inhibitor, which has been completed Phase II clinical trial for the treatment of autoimmune diseases; AIMab7195, which is in development and commercialization stage. Its product candidates also comprise XmAb14045, a bispecific antibody which is in Phase I clinical trial for the treatment of acute myeloid leukemia; plamotamab (XmAb13676) that is in Phase I clinical trial to treat B-cell malignancies; and XmAb18087, which is in Phase I clinical trial for neuroendocrine tumors and gastrointestinal stromal tumors. In addition, the company offers Tafasitamab, an antibody drug candidate for the treatment of patients with relapsed/refractory diffuse; AMG424, a bispecific antibody that targets CD38 and CD3, which is in Phase I clinical trial for the treatment of various myeloma; and AMG509, a bispecific antibody that is in preclinical development stage to treat prostate cancer. Further, it is developing bispecific antibodies to treat various cancers, such as XmAb20717, XmAb22841, and XmAb23104 which are in preclinical Phase. Additionally, it offers XmAb24306, initial cytokine candidate which is in Phase I clinical trial. The company has collaboration and license agreement with Novartis Institutes for BioMedical Research, Inc.; Amgen Inc.; Catalent Pharma Solutions LLC.; Janssen Biotech, Inc.; MorphoSys AG; Incyte Corporation; and The University of Texas MD Anderson Cancer Center. Xencor, Inc. was founded in 1997 and is headquartered in Monrovia, California.