Adeno-associated virus gene therapy delivering shortened micro-dystrophin for Duchenne muscular dystrophy.

Sarepta Therapeutics, a US-based biopharmaceutical firm, specializes in medical research and drug development.
Sarepta Therapeutics, founded in Cambridge, Massachusetts, is a commercial-stage biopharmaceutical company dedicated to the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for treating rare diseases. The company's core mission is to develop innovative treatments for patients with severe and life-threatening genetic disorders. Sarepta's product portfolio includes EXONDYS 51 and VYONDYS 53, which are designed to treat Duchenne muscular dystrophy (DMD) in patients with specific genetic mutations.
Notable figures associated with Sarepta include gene therapy pioneer Jerry R. Mendell, M.D., who was named to the inaugural TIME100 Health list of the most influential people in global health. The company has achieved significant milestones, including the successful commercialization of its DMD treatments and ongoing advancements in gene therapy research. Sarepta's impact is profound, offering hope and potential life-changing treatments to patients with rare genetic diseases, while also promoting diversity and inclusion within its organizational culture.
Companies similar to Sarepta Therapeutics include SonoThera, Solid Biosciences, PTC Therapeutics and Biogen.
Sarepta Therapeutics was founded in 1980.
Sarepta Therapeutics' headquarters is located in Cambridge, MA, US.
Sarepta Therapeutics' most recent funding round was for $6M (USD) in September 2022.
As of Sep 14, 2022, Sarepta Therapeutics has raised a total of $1.4B (USD).
Sarepta Therapeutics has 1,162 employees as of Feb 6, 2024.
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