Sarepta Therapeutics is a global biotechnology company focused on developing precision genetic medicines to treat rare diseases that significantly impact lives. The company has established a strong position in the treatment of Duchenne muscular dystrophy (DMD) and is advancing gene therapies for limb-girdle muscular dystrophy (LGMD), Charcot-Marie-Tooth (CMT) disease, MPS IIIA, and other central nervous system (CNS) disorders. Sarepta operates in the biopharmaceutical market, targeting patients with rare genetic conditions that have limited treatment options. The company's business model revolves around research and development (R&D) of innovative therapies, progressing them through clinical trials, and obtaining regulatory approvals to bring these treatments to market. Revenue is primarily generated through the sale of approved therapies and strategic partnerships. Sarepta's mission is to expedite the drug development process, reducing the time from laboratory research to patient treatment, and to build the largest gene therapy manufacturing capacity to meet global demand.

Keywords: biotechnology, genetic medicine, rare diseases, Duchenne muscular dystrophy, gene therapy, limb-girdle muscular dystrophy, Charcot-Marie-Tooth, CNS disorders, clinical trials, biopharmaceutical.