Sarepta

Sarepta is a commercial-stage biotechnology company focused on precision genetic medicines for rare diseases, with a major concentration in neuromuscular disorders. The company markets four FDA-approved therapies in the United States for Duchenne muscular dystrophy: Elevidys, a gene therapy, and the exon-skipping RNA therapies Exondys 51, Vyondys 53, and Amondys 45. Beyond its marketed Duchenne franchise, Sarepta is advancing a pipeline across gene therapy, RNA, and gene editing platforms, including programs in limb-girdle muscular dystrophy and several siRNA programs for neuromuscular and other serious diseases. Sarepta positions itself as a leader in rare-disease genetic medicine, combining internal process development and analytics capabilities with external large-scale manufacturing partnerships to support commercialization and pipeline expansion.