Sarepta Therapeutics, Inc.

Sarepta Therapeutics is a Cambridge biotechnology company that develops precision genetic medicines for rare neuromuscular and central nervous system diseases. Its work includes Duchenne muscular dystrophy, where it is studying the gene therapy ELEVIDYS (delandistrogene moxeparvovec). The company describes its mission as engineering genetic medicine to reclaim futures cut short by rare disease, with the goal of shortening the time it takes for treatments to move from the lab to patients.

Sarepta has set out to build the world’s largest gene therapy manufacturing capacity and works with health networks and payers to rethink how new genetic treatments are priced and reimbursed. Of as many as 7,000 rare diseases, only a small percentage have treatments, which the company cites as the reason for its urgency. It also supports patient communities with educational resources, advocacy programs and corporate giving, including support for investigator-initiated studies.

What to Know

• Sarepta is publicly traded on the Nasdaq.
• In 2026 it presented new ELEVIDYS data in older ambulatory Duchenne patients at the World Muscle Society congress.
• The company has locations around the world.
• Sarepta is listed with the Cambridge Chamber of Commerce.