Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company dedicated to developing and delivering transformative genetic therapies for rare diseases, with a particular emphasis on Duchenne muscular dystrophy (Duchenne). The company's core strategy revolves around RNA-targeted therapeutics and gene therapies, leveraging cutting-edge science to address the underlying genetic causes of these debilitating conditions. Sarepta's approved product portfolio includes EXONDYS 51, VYONDYS 53, and AMONDYS 45, all designed to treat specific mutations in the dystrophin gene that lead to Duchenne. Furthermore, ELEVIDYS represents a significant advancement as an adeno-associated virus-based gene therapy for ambulatory pediatric patients with Duchenne. Beyond Duchenne, Sarepta is actively expanding its pipeline, notably with its limb-girdle muscular dystrophies gene therapy program, SRP-9003. The company's business model is characterized by a strong focus on research and development, coupled with strategic collaborations and licensing agreements with leading academic institutions and industry partners to accelerate therapeutic innovation and broaden its reach. This approach allows Sarepta to maintain a leading position in the highly specialized field of rare genetic disease treatments, aiming to provide life-changing options for patients with significant unmet medical needs.