Sarepta Therapeutics 

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, siRNA platform, gene therapy, and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping; AMONDYS 45 for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping; and ELEVIDYS, an AAV-based gene therapy, which is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the Duchenne gene. The company also develops SRP-9003, a gene therapy program for the treatment of LGMD2E; SRP-1001 to selectively target and knockdown DUX4 using RNAi in Phase 1/2a clinical trials; and SRP-1003 for reduction of expression of the DMPK gene in Phase 1/2a clinical trials. It has collaboration and license agreements with F. Hoffman-La Roche Ltd; Arrowhead Pharmaceuticals, Inc.; University of Western Australia; Nationwide Children's Hospital; Hansa Biopharma; and Duke University. The company was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.