Description

Sarepta Therapeutics, Inc. operates as a biopharmaceutical firm with products already available on the market. Its primary objective is the research and advancement of genetic treatment modalities, encompassing RNA-targeted therapeutics and gene therapies, specifically for the management of rare diseases. The company's commercial offerings include EXONDYS 51, an injectable medication prescribed for Duchenne muscular dystrophy (DMD) patients whose genetic mutation in the dystrophin gene is responsive to exon 51 skipping. Similarly, VYONDYS 53 is provided for individuals with DMD who can benefit from exon 53 skipping. Sarepta is also actively developing several investigational therapies: AMONDYS 45, a product candidate that utilizes phosphorodiamidate morpholino oligomer chemistry and exon-skipping technology to bypass exon 45 of the dystrophin gene. SRP-5051, a peptide-conjugated PMO engineered to bind to exon 51 of dystrophin pre-mRNA. SRP-9001, a gene therapy initiative focused on micro-dystrophin for DMD. SRP-9003, a gene therapy program targeting limb-girdle muscular dystrophies. The company has established collaborative relationships with various partners, including F. Hoffman-La Roche Ltd, Nationwide Children's Hospital, Lysogene, Duke University, Genethon, and StrideBio. Sarepta was incorporated in 1980 and maintains its corporate headquarters in Cambridge, Massachusetts.