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RGNX vs SRPT

REGENXBIO Inc. and Sarepta Therapeutics,, Inc. Common Stock

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REGENXBIO Inc.

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 III clinical trial for the treatment of Duchenne muscular dystrophy, as well as RGX-121 for the treatment of mucopolysaccharidosis type II. It also develops RGX-111 for the treatment of mucopolysaccharidosis type I, RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2, and RGX-381 for the treatment of 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.

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Sarepta Therapeutics,, Inc. Common Stock

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 that is amenable to exon 45 skipping; and ELEVIDYS, an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients with Duchenne with a confirmed mutation in the Duchenne gene. The company is also developing SRP-9003, a limb-girdle muscular dystrophies gene therapy program. It has collaboration and license agreements with F. Hoffman-La Roche Ltd; Arrowhead Pharmaceuticals, Inc.; University of Western Australia; Catalent Maryland, Inc.; Nationwide Children's Hospital; Dyno Therapeutics; Hansa Biopharma; Duke University; and Genethon. The company was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.

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