Sarepta Therapeutics Inc is a biotechnology company that specializes in the development of precision genetic medicines for the treatment of rare diseases. The company was founded in 1980 under the name AntiVirals Inc. It later changed its name to AVI BioPharma and then to Sarepta Therapeutics in 2012. The company's headquarters are located in Cambridge, Massachusetts, USA.
Sarepta Therapeutics focuses on developing therapies for rare genetic diseases that are caused by specific genetic mutations. Their primary area of expertise is in developing treatments for Duchenne muscular dystrophy (DMD), a severe and progressive muscle-wasting disease that primarily affects boys. DMD is caused by mutations in the dystrophin gene, and Sarepta has been at the forefront of research and development in this field.
The company's most notable drug is Exondys 51 (eteplirsen), which is the first FDA-approved treatment for a specific subset of DMD patients with a particular genetic mutation. Exondys 51 aims to promote the production of a shortened but functional form of dystrophin, the protein missing or insufficiently produced in DMD patients.