List of Adeno-associated viral vector (AAV) Companies in United States - 244

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.

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.

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.

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.

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.

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.

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

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

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.

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.

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

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.

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.

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.

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.

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.

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

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.

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.

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.

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.

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.

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.

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.

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.

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 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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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/ .

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.

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.

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.

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,

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.

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.

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

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.

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

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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

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.

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.

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.

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.

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.

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 focuses on healthcare education and medical research.

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.

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.

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.

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.

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.

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).

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.

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.

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.