Sarepta Therapeutics is a clinical-stage biopharmaceutical company specializing in the discovery and development of innovative genetic medicines for rare neuromuscular diseases. Headquartered in Cambridge, Massachusetts, the company’s core focus lies in designing and commercializing precision therapies that address the underlying genetic causes of disorders such as Duchenne muscular dystrophy (DMD). Through its proprietary exon-skipping and gene therapy platforms, Sarepta seeks to deliver targeted treatments that modify disease progression rather than solely providing symptomatic relief.
The company’s commercial portfolio includes FDA-approved exon-skipping therapies for DMD, notably Exondys 51 (eteplirsen), Vyondys 53 (golodirsen) and Amondys 45 (casimersen). These synthetic antisense oligonucleotides are designed to restore the dystrophin protein in patients with specific genetic mutations. In parallel, Sarepta is advancing its gene therapy programs, including Elevidys (SRP-9001) for DMD and SRP-9003 for limb-girdle muscular dystrophy type 2E. These adeno-associated virus (AAV)–based candidates represent a one-time treatment approach aimed at delivering functional copies of disease-modifying genes to muscle tissue.
Founded through the evolution and rebranding of earlier research efforts in antisense technology, Sarepta has emerged as a leader in the field of genetic medicine since adopting its current name in 2016. The company is led by President and Chief Executive Officer Douglas Ingram, whose background spans both commercial and clinical development in rare diseases. Under his leadership, Sarepta has expanded its global footprint, establishing collaborations with regulatory authorities and academic institutions in North America, Europe and Asia to support clinical trials and regulatory submissions.
Looking ahead, Sarepta continues to enrich its pipeline with next-generation platforms, including micro-dystrophin gene therapy, peptide-conjugated PMOs and gene editing approaches. The company maintains strategic partnerships with industry peers and research organizations to enhance manufacturing capabilities and accelerate global patient access. By combining precision molecular science with a patient-centric development model, Sarepta aims to transform the treatment landscape for individuals affected by rare neuromuscular conditions.
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