Sarepta Therapeutics Inc.

Sarepta Therapeutics Inc. is a commercial-stage biopharmaceutical company focused on the discovery, development, and commercialization of precision genetic medicines for rare diseases. It specializes in RNA-targeted therapeutics, gene therapies, and other genetic modalities, primarily addressing Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies. Key products include EXONDYS 51 injection for DMD patients amenable to exon 51 skipping, VYONDYS 53 for exon 53 skipping, AMONDYS 45 for exon 45 skipping, and ELEVIDYS, an adeno-associated virus-based gene therapy for ambulatory pediatric patients with confirmed Duchenne gene mutations. The company advances a pipeline of investigational therapies targeting additional rare neuromuscular and central nervous system disorders. Operating in the health technology sector within pharmaceuticals, Sarepta Therapeutics Inc. plays a critical role in providing innovative treatments for patients with limited options, emphasizing exon-skipping technologies and gene therapy platforms to address underlying genetic causes. Founded in 1980 and headquartered in Cambridge, Massachusetts, it supports clinical research and commercial efforts to expand access to these specialized therapies.