Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for 6 Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases.

OPTIMIZED TO GET TO THE HEART OF DMD

To get to the heart of DMD, it is critical for gene therapy to be delivered to skeletal and cardiac muscle.

Our goal is to develop therapies to treat 100% of individuals with this devastating disease.

Our commitment to the patients and families impacted by Duchenne muscular dystrophy extends beyond treatment. We are proud to offer SareptAssist™, a patient support program for those seeking information on therapy.

Our unique RNA-based technology has the potential to advance treatments in numerous areas of high unmet need. We are continuing to advance our clinical programs for Duchenne muscular dystrophy.

We're looking for people who exhibit teamwork, excellence, innovation, drive, compassion, and resilience. If you thrive in an environment where you can make a real difference in people's lives and where you're challenged to be the best that you can be, we encourage you to come be part of our mission.

We seek collaborations with leaders in industry, government, and academia to make meaningful advances in medicine. Our proprietary and highly versatile RNA-based technologies form the foundation of our partnering strategy.