408 Adeno-associated Viral Vector (AAV) Companies - Worldwide
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Company Name | Location | About |
|---|---|---|
Malmö | 2A Pharma is a clinical stage Swedish/Danish biopharmaceuticals company focused on discovering, developing and commercialising novel, cost effective prophylactic and therapeutic vaccines based on our patented vaccine platform. Our lead vaccine candidate, 2AP01, is a HPV (human papillomavirus) vaccine formulated without adjuvant. We believe 2AP01 will offer significant advantages over existing vaccines including broader protection against more HPV types and lower production cost. Our vaccine technology is based on adeno-associated virus-like particles (AAVLPs), that assemble viral capsids without containing viral genomes; hence they are replicative-defective and non-pathogenic. The viral capsid has repetitive immunogenic sites that can be modified to encompass antigenic epitopes for a protein of interest, generating an immune response where both the innate and the adaptive immune system are activated. In other words, 2A Pharma exploit the efficacy of the individuals’ own immune system to generate AAVLP-based vaccines that, without the addition of adjuvants, are able to develop highly effective antibodies with selected specificities and functions. The technology enables the production of highly efficient vaccines with significant competitive advantages | |
Cambridge, GB | 4basebio is enabling next generation cell and gene therapies and vaccines with its technologies and solutions. We are able to design, manufacture and supply application-specific synthetic DNA or mRNA as well as targeted non-viral vectors for the delivery of nucleic acid payloads. Our novel synthetic DNA technology offers unique customisation potential, rapid turn-around times, and improved safety profiles, addressing the current limitations of plasmids and other DNA technologies. We currently offer four DNA construct types, each ideally suited for use in viral and non-viral vector applications, genome editing, vaccines and therapeutics, and DNA vaccine applications, respectively. Our proprietary non-viral delivery system, Hermes™, is a nanoparticle vector that can be customised to target cells or tissues of interest for a range of applications. This combines the safety and efficiency of lipid-based nanoparticles (LNPs) with the specificity of a targeting system, addressing the shortcoming associated with both viral vectors and non-targeted LNPs. | |
 | Emeryville, California, United States of America | 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. |
Heidelberg, Baden-Württemberg, Germany | AaviGen GmbH is a biotechnology start-up located in Heidelberg, Germany. Our primary purpose is the development and commercialization of precision gene therapies for cardiovascular and cardiopulmonary diseases. Our business model builds on the success of the InoCard GmbH, a predecessor that was acquired by uniQure N.V. and subsequently entered into a development alliance with Bristol-Myers-Squibb. Please find our imprint ("Impressum") and data privavy statement ("Datenschutzerklärung") at https://aavigen.com/content/imprint.html and https://aavigen.com/content/privacy.html | |
North Bethesda, Maryland, US | 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. | |
Washington, United States | 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. | |
Rockville, Maryland, United States of America | 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. | |
New York, New York, United States of America | 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. | |
San Diego, California, United States | 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. | |
Cranbury, New Jersey, US | ||
Raleigh, North Carolina | 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. | |
Cary, North Carolina, United States | Advanced Medicine Partners’ aims to set a new standard for manufacturing and testing innovative medicines that can potentially transform the lives of patients and families. The company delivers process development, manufacturing, analytical development and testing for advanced therapy products, including gene therapies. The company has the experience and capabilities to work with multiple platforms and modalities, including adeno-associated virus (AAV), lentivirus and other delivery systems. Advanced Medicine Partners leverages one of the most experienced genetic medicines teams – at all levels, from bench to boardroom – in the industry, and its exclusive manufacturing platform consistently produces yields that are among the highest in the industry and purity profiles that push the boundaries of what’s possible. The company’s proprietary adeno-associated virus (AAV) manufacturing process minimizes process and product-related impurities and consistently generates industry-leading functional full capsid ratios and best-in-class yields. This manufacturing process prioritizes patient safety while reducing cost of goods and will strive to set the standard for what regulatory agencies expect and patients deserve. Advanced Medicine Partners brings extensive operational experience from Jaguar Gene Therapy and other biotechnology companies. The Advanced Medicine Partners team has manufactured upwards of 350 total non-GMP batches and supplied over 20 preclinical studies, including initial new drug application (IND) enabling efficacy and good laboratory practice (GLP) toxicology studies. The company distinguishes itself from CDMOs by its partnership philosophy. Traditionally, CDMO operations work independently of their clients. Advanced Medicine Partners changes that dynamic by acting as a client’s extended workbench, removing CMC activities from the critical path, and offering real-time data to help clients drive decisions and accelerate their programs. | |
Redwood City, California, United States of America | 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. | |
Oak Park, Illinois, United States | The company is developing onlytic immunotherapies for treating cancer. | |
Waltham, Massachusetts | 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. | |
Fargo, North Dakota, US | 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 visit | |
22021 20th Avenue SE, Bothell, WA 98021, US | 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. | |
Alameda, California, United States | 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. | |
Alachua, Florida, United States | 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. | |
Itasca, Illinois, United States | 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. | |
Abingdon, Oxfordshire, United Kingdom | Akamis is a clinical-stage oncology company whose mission is to leverage its groundbreaking T-SIGn® platform to positively impact the lives of people living with cancer. We are developing a portfolio of solid tumor-targeted T-SIGn® therapeutics which aim to enable a patient’s own immune system to recognize, attack, and clear their cancer. Akamis’ growing pipeline of T-SIGn® therapeutics is anchored by its lead clinical-stage program, NG-350A, which is being investigated in an ongoing Phase 1 clinical study in patients with metastatic or advanced epithelial tumors. Akamis has a number of T-SIGn® platform-focused collaborations with leaders in the immuno-oncology field including BMS, Merck, and the Parker Institute for Cancer Immunotherapy (PICI) | |
Boston, Massachusetts, United States of America | 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. | |
Lowell, Massachusetts, United States | 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). | |
Boston, Massachusetts, United States | 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. | |
Trento, IT | Alia Therapeutics is a pioneering gene editing company with a vision to revolutionize medicine by curing genetic diseases at their roots. Alia’s versatile portfolio of novel CRISPR systems expands the range of targetable genomic sites and increases the precision of genetic modifications, bringing gene editing to the next level of specificity and safety, which are required for therapeutic applications. | |
Vancouver, British Columbia, Canada | Alpha Cognition is a clinical stage, biopharmaceutical company dedicated to developing novel treatments for under-served neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis (ALS). ALPHA-1062, a patented new chemical entity that has demonstrated safety and improved tolerability in human clinical trials. It is being developed as a new acetylcholine esterase inhibitor for the treatment of dementia of the Alzheimer’s type, with minimal gastrointestinal side-effects and novel routes of administration. ALPHA-0602 is a gene therapy program delivering progranulin, a neurotrophic protein. It is in preclinical development for the treatment of ALS. ALPHA-0602 is patented and has received Orphan Drug Designation from the FDA. | |
Hollister, California, 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. | |
Leiden, Netherlands | Amarna Therapeutics is a privately held, pre-clinical biotech company developing transformative, potentially curative gene therapies for a range of both rare and prevalent diseases. The company is pioneering the development of gene therapies based on simian virus 40 (SV40), a macaque polyoma virus to which humans are immunologically naïve. Amarna has created a proprietary production cell line (SuperVero™) that, for the first time, makes it possible to produce SV40-derived vectors suitable for therapeutic use. Combining SuperVero™ with its genetically engineered SVec viral vector, the company’s fully-integrated gene therapy platform is truly unique in its intrinsic capability to deliver transgenes without eliciting immune responses to the vector nor to the transgene product. Leveraging this ‘natural’ non-immunogenicity feature of its gene delivery system in humans, Amarna is developing a broad pipeline of safe, effective and durable gene therapies focused on genetic disorders, autoimmune diseases and chronic inflammation. Founded in 2008, Amarna’s head office is based in Leiden Bio Science Park, the Netherlands, with a research subsidiary located in Seville, Spain. | |
Miami, Florida, United States of America | 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. | |
Philadelphia, Pennsylvania, United States of America | 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. | |
Towson, Maryland, United States of America | Amplo Biotechnology is a private Gene Therapy company focusing on therapy for diseases of the neuromuscular junction. | |
Columbus, Ohio, United States | 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. | |
10 Anxiang Street, Shunyi, Beijing | At Anlong Bio, patients are our priority. Since launching, our goal has been to deliver transformative gene therapy medicines to patients faster and at low cost, in China and globally. We aim to provide unmatched access to treatments for patients through our extensive resources, our experienced leadership and advisory teams, and our trusted network of partners. | |
Davis, California, United States | Antibodies, Assays, Development Services, Diagnostic kits,etc | |
New York, New York | 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. | |
Fremont, California, United States | custom-made recombinant viral vectors | |
Cambridge, Massachusetts, US | 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. | |
Tromso, Norway | ArcticZymes Technologies ASA (OSE: AZT) is a global leader in supplying best-in-class enzyme technologies to commercial partners who develop therapeutics, in vitro diagnostics, and molecular research kit products. Our mission is to make our customers lives easier by unlocking new solutions to enhance quality of life and create a more sustainable healthy world. We provide our customers with cold-adapted enzymes from the arctic environment with unique features including heat lability and activity in challenging environments, as well as, customised engineered novel features which can be easily integrated to fuel their innovations. For more information, please visit www.arcticzymes.com | |
987 old eagle school rd, tromsø, troms og finnmark, norway | ArcticZymes Technologies ASA (OSE: AZT) is a global leader in supplying best-in-class enzyme technologies to commercial partners who develop therapeutics, in vitro diagnostics, and molecular research kit products. Our mission is to make our customers lives easier by unlocking new solutions to enhance quality of life and create a more sustainable healthy world. We provide our customers with cold-adapted enzymes from the arctic environment with unique features including heat lability and activity in challenging environments, as well as, customised engineered novel features which can be easily integrated to fuel their innovations. For more information, please visit www.arcticzymes.com | |
Columbus, Ohio, US | Armatus Bio is a biotechnology company that specializes in precision gene therapy for raising expectations in FSHD. | |
London, United Kingdom | Ascend is a development partner that supports life-science innovators in bringing gene therapy products to patients. Our team has deep expertise in gene therapy development and commercialization to offer unparalleled collaboration while working to develop your products from the bench through the clinic and beyond. Scientific innovation driven by quality and responsibility are at the core of our business. To set a new standard in the industry, we have invested in a world-class analytical toolkit within our flexible and scalable AAV platform of next-gen technologies, all built to find the perfect balance of yield and quality for each process. Let’s aim higher and develop the next wave of accessible life-saving therapies together! | |
Alameda, California, United States | The company supports cell and gene therapy manufacturing and process development projects from design through to clinical and commercial scale. | |
Milpitas, California, United States | 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 | |
Lund, Sköna, Sweden | Asgard Therapeutics is a private biotech company exploring the application of direct cell reprogramming technologies for cancer immunotherapies. Formed as a spin-off from Lund University, the Company is pioneering a gene therapy approach based on its proprietary TrojanDC technology, designed to set in motion immune responses based on the biological properties of professional antigen presenting cells. Backed by Novo Holdings, Boehringer Ingelheim Venture Fund and Industrifonden, Asgard Therapeutics aims to build a pipeline of personalized cancer immunotherapies optimized to each unique patient. | |
Boston, Massachusetts, US | 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. | |
Chapel Hill, North Carolina, United States | 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. | |
 | Palo Alto, California, United States of America | 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. |
Northbrook, Illinois, United States | 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. | |
Cambridge, United Kingdom | Driven by innovative science and our entrepreneurial culture, we are focused on the delivery of life-changing medicines that are fuelling growth and contributing value to patients and society. | |
Evry, France | Atamyo Therapeutics is focused on the development of a new generation of safe and effective gene therapies for neuromuscular diseases. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD). |