Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases.

Sarepta is a global biotechnology company on an urgent mission: to engineer precision genetic medicine for rare diseases that devastate lives and cut futures short.

Jun 27, 2024 · Sarepta’s industry leading pipeline is comprised of over 40 programs in various stages of development across 3 technologies, RNA, gene therapy and gene editing, and multiple therapeutic areas including neuromuscular, CNS and cardiology.

Learn about Sarepta's leadership and their goal to forever change the course of genetic disease.

Read news and articles from Sarepta, a global biotechnology company developing potentially life-changing precision genetic medicine.

Sarepta currently has multiple clinical trials for Duchenne underway in both gene therapy and RNA exon-skipping.

We’re looking for people who see unlimited potential in themselves, backed by an unwavering commitment to patients. Learn more about careers at Sarepta.

Dec 18, 2024 · LGMD 2C/R5: Study SRP-9005-101, Sarepta’s first human study of an investigational gene therapy designed for people living with LGMD2C/R5, is expected to initiate in early 2025. Sarepta looks forward to sharing further updates about the program when they become available.

At Sarepta, we’re committed to pursuing some of the world’s most debilitating, prevalent, and complex rare genetic diseases. Today, our primary focus is on Duchenne muscular dystrophy, limb-girdle muscular dystrophies, and Charcot-Marie-Tooth disease.

Jun 22, 2023 · CAMBRIDGE, Mass. -- (BUSINESS WIRE)--Jun. 22, 2023-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus …