Sarepta Therapeutics, Inc. is a biopharmaceutical company focused on the discovery and development of precision genetic medicine for rare neuromuscular diseases. Leveraging expertise in RNA-targeted therapeutics and gene therapy, Sarepta designs and develops treatments that aim to address the underlying genetic causes of diseases such as Duchenne muscular dystrophy (DMD) and other serious rare disorders. The company’s research platform encompasses exon skipping, gene replacement, and gene editing approaches to advance novel therapies from early research through commercialization.
The company’s most advanced products include RNA-based exon-skipping therapies that modulate the expression of dystrophin in patients with specific DMD mutations. In addition to its marketed therapies, Sarepta’s pipeline features a broad array of next-generation gene therapy candidates delivered via adeno-associated virus vectors, targeting systemic delivery to muscle tissue. Preclinical and clinical programs also explore genome editing technologies and microdystrophin constructs designed to improve safety, efficacy and durability of treatment outcomes.
Founded in 1980 as AVI BioPharma and rebranded as Sarepta Therapeutics in 2016 following a strategic merger and corporate transformation, the company established its global headquarters in Cambridge, Massachusetts. Over the years, Sarepta has formed strategic collaborations with academic institutions and industry partners to accelerate research in neuromuscular disease and expand its manufacturing capabilities. The company also maintains commercial and clinical operations in Europe and Asia, working closely with patient advocacy groups and regulatory agencies to facilitate access to innovative therapies.
Since 2022, Sarepta has been led by President and Chief Executive Officer Douglas L. Ingram, who brought extensive experience in rare disease drug development and commercialization to the organization. Under his leadership, the company continues to invest in next-generation genetic medicine platforms, bolster its manufacturing network and pursue regulatory approvals that could extend therapies to broader patient populations. Sarepta’s commitment to scientific innovation and patient-focused development has positioned it as a key player in the evolving field of precision medicine for neuromuscular disorders.
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