Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company that focuses on the discovery, development, and commercialization of precision genetic medicines for rare diseases. The company's core expertise lies in RNA-targeted therapeutics, gene therapy, and gene editing modalities. Sarepta's primary therapeutic focus is on Duchenne muscular dystrophy (DMD), a rare and fatal genetic disorder. The company has built a leading franchise in DMD, with a portfolio of approved therapies that includes ELEVIDYS, a one-time gene therapy, as well as several RNA-based exon-skipping drugs such as EXONDYS 51, VYONDYS 53, and AMONDYS 45. Revenue is primarily generated from the sale of its approved DMD therapies in the United States, alongside collaboration and milestone revenues from partnerships, such as its agreement with Roche for the commercialization of ELEVIDYS outside the U.S. The company operates primarily in the United States but has a growing international footprint through strategic partnerships. Sarepta's competitive position is defined by its pioneering role in genetic medicine and its robust pipeline of candidates targeting various rare neuromuscular, neurological, and cardiomyopathic diseases, including limb-girdle muscular dystrophies (LGMD). The company continues to invest heavily in research and development to expand its genetic medicine platform and address unmet medical needs in rare diseases.
