Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company focused on discovering and developing RNA-targeted therapeutics, siRNA platforms, gene therapy, and other genetic therapeutic modalities for rare diseases. Its approved products include EXONDYS 51, VYONDYS 53, and AMONDYS 45, which are used to treat Duchenne muscular dystrophy in patients with specific genetic mutations amenable to exon 51, 53, or 45 skipping. The company also offers ELEVIDYS, an AAV-based gene therapy for Duchenne, and has a pipeline including SRP-9003 for LGMD2E, as well as SRP-1001 and SRP-1003 for other rare conditions. Sarepta collaborates with organizations such as F. Hoffman-La Roche Ltd, Arrowhead Pharmaceuticals, and Nationwide Children's Hospital.

Sarepta Therapeutics is listed on the Nasdaq Global Select Market (NasdaqGS) under the ticker symbol SRPT. Its shares are traded in US dollars (USD), as the company is headquartered in the United States and reports its financial results in USD. The exchange is a major US stock exchange, providing liquidity and visibility for investors.

Sarepta Therapeutics operates in the Healthcare sector, specifically within the Biotechnology industry. As a biotechnology company, it focuses on developing innovative therapies for rare genetic diseases, leveraging technologies such as RNA-targeted therapeutics, gene therapy, and siRNA platforms. Its primary area of expertise is in neuromuscular disorders, particularly Duchenne muscular dystrophy and limb-girdle muscular dystrophy.

Sarepta Therapeutics is headquartered in Cambridge, Massachusetts, United States. The company primarily serves the US market, as its approved products (EXONDYS 51, VYONDYS 53, AMONDYS 45, and ELEVIDYS) are available for patients in the US. Through collaborations with partners like F. Hoffman-La Roche Ltd, it may also expand its reach to international markets. The company focuses on rare diseases, targeting patient populations with specific genetic mutations.

Sarepta's main commercial products include EXONDYS 51, VYONDYS 53, and AMONDYS 45, which are exon-skipping therapies for Duchenne muscular dystrophy, and ELEVIDYS, a gene therapy for Duchenne. Its pipeline includes SRP-9003, a gene therapy for LGMD2E, and SRP-1001 and SRP-1003, which are RNAi-based therapies targeting DUX4 and DMPK, respectively, currently in Phase 1/2a trials. The company also has collaboration agreements with F. Hoffman-La Roche Ltd, Arrowhead Pharmaceuticals, University of Western Australia, Nationwide Children's Hospital, Hansa Biopharma, and Duke University to advance its research and development.