SRPT

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.

Funcionários

835

Sede

Cambridge, United States

Site oficial

sarepta.com

Descrição da empresa fornecida pelo Yahoo Finance. A descrição é publicada pela fonte apenas em inglês.