Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping; AMONDYS 45 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene; and ELEVIDYS, an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with duchenne with a confirmed mutation in the duchenne gene. The company is also developing SRP-5051, a peptide conjugated PMO that binds exon 51 of dystrophin pre-mRNA; and SRP-9003, a limb-girdle muscular dystrophies gene therapy program. It has collaboration and license agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Genevant Sciences; University of Florida; Dyno Therapeutics; Hansa Biopharma; Duke University; Genethon; and StrideBio. The company was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.

Ocular Therapeutix, Inc., a biopharmaceutical company, focuses on the formulation, development, and commercialization of therapies for diseases and conditions of the eye using its bioresorbable hydrogel-based formulation technology in the United States. The company markets DEXTENZA, a dexamethasone ophthalmic insert to treat post-surgical ocular inflammation and pain following ophthalmic surgery, as well as allergic conjunctivitis. It is developing AXPAXLI, an axitinib intravitreal implant that is in phase 3 trials for the treatment of wet age-related macular degeneration and other retinal diseases; PAXTRAVA, a travoprost intracameral implant, which is in phase 2 clinical trials for the treatment of open-angle glaucoma or ocular hypertension; OTX-CSI, a cyclosporine intracanalicular insert that has completed phase 2 clinical trials for the treatment of dry eye disease; and OTX-DED, a dexamethasone intracanalicular insert, which is in phase 2 clinical trials for the short-term treatment of the signs and symptoms of dry eye disease. In addition, the company offers modulator for intermediate and late dry age-related macular degeneration; and gene delivery for inherited retinal degenerations and protein biofactory indications. The company has a strategic collaboration with Regeneron Pharmaceuticals, Inc. (Regeneron) for the development and commercialization of products using the company's sustained-release hydrogel in combination with Regeneron's large molecule VEGF-targeting compounds for the treatment of retinal diseases; and AffaMed Therapeutics Limited for the development and commercialization of DEXTENZA and OTX-TIC. Ocular Therapeutix, Inc. was incorporated in 2006 and is headquartered in Bedford, Massachusetts.

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase I/II clinical trial for the treatment of Duchenne muscular dystrophy. It also develops RGX-121 for the treatment of mucopolysaccharidosis type II that is in Phase III clinical trial; RGX-111 for treating mucopolysaccharidosis type I; RGX-181 for the treatment of late infantile neuronal ceroid lipofuscinosis type II; and RGX-381 to treat the ocular manifestations of CLN2 disease. In addition, the company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. Further, it has a collaboration and license agreement with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States. REGENXBIO Inc. was incorporated in 2008 and is headquartered in Rockville, Maryland.

Neurogene Inc., a biotechnology company, develops genetic medicines for rare neurological diseases. The company's product candidates include NGN-401 which is packaged in an adeno-associated virus 9 that is in Phase 1/2 clinical trial for the treatment of Rett syndrome; and NGN-101, a conventional gene therapy candidate that is in Phase 1/2 clinical trial to treat CLN5 Batten disease. It has a license agreement with The University of North Carolina, the University of Edinburgh, Virovek, Inc., and Sigma-Aldrich Co. LLC. The company is headquartered in New York, New York.

Ocugen, Inc., a clinical-stage biopharmaceutical company, focuses on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve patients' health. The company's pipeline product includes OCU400, a novel gene therapy product candidate restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases, currently under Phase 3 trials for the treatment of retinitis pigmentosa and Phase 1/2 trials for the treatment of leber congenital amaurosis; OCU410, a gene therapy under phase 1/2 for the treatment of dry age-related macular degeneration (AMD); and OCU410ST, a gene therapy under phase 1/2 for the treatment of Stargardt disease. It is also involved in the development of OCU200, a novel fusion protein that is in preclinical development stage for the treatment of diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration; and NeoCart, an autologous chondrocyte-derived neocartilage, currently under Phase 3 studies indicated for the repair of knee cartilage injuries in adult. In addition, the company is developing OCU500, a COVID-19 vaccine; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine. It has collaboration agreements with National Institute of Allergy and Infectious Diseases for early clinical studies for the OCU500 program; and a strategic partnership with CanSino Biologics Inc. for manufacturing its modifier gene therapy pipeline product candidates. The company was founded in 2013 and is headquartered in Malvern, Pennsylvania.

4D Molecular Therapeutics, Inc., a clinical-stage biotherapeutics company, develops genetic medicines using its therapeutic vector evolution platform in the Netherland and the United States. The company develops a portfolio of genetic medicine product candidates focuses in three therapeutic areas for ophthalmology, cardiology, and pulmonology. Its product pipeline includes 4D-150, which is in phase 1/2 clinical trial for the treatment of wet age-related macular degeneration; and is in phase 1/2 clinical trial for the treatment of diabetic macular edema. The company develops 4D-125, which is in phase 1/2 clinical trial for the treatment of x-linked retinitis pigmentosa; 4D-110, which is in phase 1/2 clinical trial for the treatment of choroideremia; 4D-710, which is in phase 1/2 clinical trial for the treatment cystic fibrosis lung disease; and 4D-310 to treat fabry disease cardiomyopathy and is in phase 1/2 clinical trial. In addition, its product candidates comprises 4D-175, which is in pre-clinical development for the treatment for geographic trophy; and 4D-725 to treat alpha-1 antitrypsin deficiency lung disease and is in pre-clinical development. The company has collaboration and licensing agreements with Arbor Biotechnologies, Inc.; Astellas Gene Therapies, Inc; uniQure biopharma B.V.; and Cystic Fibrosis Foundation. 4D Molecular Therapeutics, Inc. was founded in 2013 and is headquartered in Emeryville, California.

Lexeo Therapeutics, Inc. operates as a clinical stage genetic medicine company that focuses on hereditary and acquired diseases. The company develops LX2006, which is an AAVrh10-based gene therapy candidate for the treatment of Friedreich's ataxia (FA) cardiomyopathy; LX2020, an AAVrh10-based gene therapy candidate for the treatment of plakophilin-2 arrhythmogenic cardiomyopathy; LX2021, a gene therapy candidate for the treatment of DSP cardiomyopathy associated with it; and LX2022, a gene therapy candidate for the treatment of hypertrophic cardiomyopathy, or HCM caused by TNNI3 gene. It also develops LX1001, an AAVrh10-based gene therapy candidate for the treatment of APOE4 homozygous; LX1020, a gene therapy candidate for the treatment of APOE4 homozygous; LX1021 for the treatment of APOE4 homozygotes; and LX1004 for the treatment of CLN2 Batten disease. The company was incorporated in 2017 and is based in New York, New York.

Adverum Biotechnologies, Inc., a clinical-stage company, develops gene therapy product candidates to treat ocular diseases. Its lead product candidate is ixoberogene soroparvovec (ADVM-022), a single intravitreal injection gene therapy candidate used for the treatment of patients with wet age-related macular degeneration and diabetic macular edema which is in phase 2 clinical trials. The company is developing ADVM-062 (AAV.7m8-L-opsin), a novel gene therapy product candidate for the treatment of blue cone monochromacy via a single IVT injection. Adverum Biotechnologies, Inc. has license and collaboration agreements with University of California; GenSight Biologics; Lexeo Therapeutics; and Virovek, Inc. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was incorporated in 2006 and is headquartered in Redwood City, California.