List of Adeno-associated viral vector (AAV) Companies in New York - 16

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.

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.

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.

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.

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

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

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.

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.

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

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).

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.

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.

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.

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.

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.