Sarepta Therapeutics, Inc. is a biopharmaceutical company focused on the discovery and development of precision genetic medicines for rare neuromuscular diseases. Headquartered in Cambridge, Massachusetts, Sarepta’s core expertise lies in designing RNA-targeted therapies and gene therapies that address underlying genetic mutations. The company’s mission is to transform the treatment paradigm for patients with Duchenne muscular dystrophy (DMD) and related disorders through innovative modalities.
Sarepta’s commercial products include several exon-skipping therapies approved by the U.S. Food and Drug Administration for specific DMD mutations. Exondys 51 (eteplirsen), Vyondys 53 (golodirsen) and Amondys 45 (casimersen) use chemically modified oligonucleotides to induce exon skipping and restore production of functional dystrophin protein. In addition, Elevidys (delandistrogene moxeparvovec) is an adeno-associated virus (AAV)-based gene therapy designed to deliver a shortened dystrophin gene to muscle tissue, representing one of the first approved gene therapies for DMD.
Founded in the late 1980s under the name AVI BioPharma and rebranded as Sarepta Therapeutics in 2012, the company initially explored antiviral technologies before pivoting to neuromuscular disease in 2010. Sarepta now maintains commercial operations across North America and has established collaborations in Europe and Asia to support clinical trials and regulatory filings. The company continues to expand its pipeline, exploring next-generation gene editing, microdystrophin constructs and treatments for other genetic muscle disorders such as limb-girdle muscular dystrophy.
Leadership at Sarepta is spearheaded by President and Chief Executive Officer Douglas Ingram, who joined the company in 2018 and assumed the CEO role in 2021. Under his guidance, Sarepta has grown from a single-product organization into a multi-asset innovator with a broad platform in precision genetic medicine. The company’s executive team combines expertise in neurology, genetic engineering and regulatory affairs to advance therapies from early-stage research through global commercialization.
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