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Sarepta makes medicines that target the genetic causes of rare muscle-wasting diseases, especially Duchenne muscular dystrophy.
Sarepta Therapeutics is a biotech company that makes medicines for a small group of people with rare genetic diseases — mostly boys with Duchenne muscular dystrophy, a brutal condition where muscles slowly break down. Duchenne happens because a gene called dystrophin is broken, so the body can't make the protein that keeps muscle cells intact. Sarepta's drugs try to patch that problem: some skip over the broken part of the gene so the body can make a shortened but still useful protein, and others aim to deliver a working version of the gene outright.
The company sells several of these "exon-skipping" drugs — names like Exondys 51, Vyondys 53, and Amondys 45 — each aimed at a different slice of Duchenne patients. These are infused into patients and priced high, because they serve tiny populations and took years of expensive science to build. In 2023, Sarepta won the first-ever approval for a gene therapy for Duchenne, called Elevidys, which delivers a working dystrophin gene in a single dose. It's the company's biggest bet and its most controversial one: the drug was approved under accelerated rules, and its confirmatory trial later fell short of its main goal, though regulators allowed it to stay on the market and even widened its label. Doctors and investors are still arguing about how well it really works.
Sarepta is based in Cambridge, Massachusetts, and it spends heavily on research — it has burned cash for years chasing these therapies. The business is high-risk, high-reward: a handful of rare-disease drugs, ultra-high prices, and heavy reliance on a few products. Its main competition comes from other Duchenne players and from newer approaches like Wave Life Sciences and Avidity Biosciences in the same space. If Elevidys delivers, Sarepta becomes a gene-therapy powerhouse; if safety or efficacy worries grow, the whole story wobbles.
Saved answer · last researched 15 Nov 2026