Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of precision genetic medicines for rare diseases, with a primary focus on neuromuscular disorders. Headquartered in Cambridge, Massachusetts, the company's mission is to engineer advanced genetic medicines to rescue lives impacted by rare diseases, striving to accelerate the translation of genetic understanding into tangible treatments.

Sarepta's product portfolio includes RNA-targeted exon-skipping therapies such as EXONDYS 51, VYONDYS 53, and AMONDYS 45, alongside its gene therapy, ELEVIDYS (delandistrogene moxeparvovec-rokl). These therapies are primarily aimed at treating Duchenne muscular dystrophy (DMD) and other neuromuscular conditions. The company also provides patient support services through programs like SareptAssist and SareptAlly.

As a leader in precision genetic medicine for rare neuromuscular disorders, Sarepta Therapeutics has seen significant developments, including the FDA's accelerated approval of ELEVIDYS in June 2023 and its subsequent label expansion in June 2024 for Duchenne muscular dystrophy. Douglas S. Ingram serves as the CEO and President. The company continues to advance its pipeline and reported substantial net product revenues, with Q1 2025 net product revenues reaching $611.5 million and full-year 2024 total net product revenue at $1.79 billion.