薩雷普塔治療 (Sarepta Therapeutics) 

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.