Sarepta Therapeutics, Inc. SRPT

SRPT Company Overview

Sarepta Therapeutics, Inc. is a U.S.-based biopharmaceutical company focused on the discovery, development, and commercialization of precision genetic medicines for rare neuromuscular diseases. The company primarily operates in the biotechnology and rare disease therapeutics industries, with a concentrated emphasis on Duchenne muscular dystrophy (DMD) and other genetically defined disorders. Sarepta’s core capabilities center on RNA-targeted therapies and gene therapy, leveraging proprietary platforms to address the underlying genetic causes of disease rather than symptoms alone.

The company’s primary revenue drivers are its FDA-approved DMD therapies, including Exondys 51, Vyondys 53, and Amondys 45, which are exon-skipping antisense oligonucleotide treatments, as well as Elevidys, a gene therapy approved for certain DMD patients. Sarepta serves a specialized patient population within the rare disease community, primarily pediatric and young adult patients, and is uniquely positioned as a market leader in DMD due to its early-mover advantage, deep clinical expertise, and vertically integrated development and manufacturing capabilities. Founded in 1980 as AVI BioPharma, the company rebranded as Sarepta Therapeutics in 2012 to reflect its strategic shift toward rare genetic diseases and precision medicine.