Sarepta Therapeutics | Biopharmaceutical Company for Rare …
Sarepta is engineering solutions for rare diseases with science that is on the forefront of precision genetic medicine. Our platforms include: gene therapy, RNA technologies, and gene editing. We are in a daily race to save lives stolen or impacted by rare disease.
About Our Company - Sarepta Therapeutics
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.
Our Pipeline | Sarepta Therapeutics
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.
News & Press Releases - Sarepta Therapeutics
Read news and articles from Sarepta, a global biotechnology company developing potentially life-changing precision genetic medicine.
The Science Behind Our Precision Genetic Medicine Platforms
Sarepta is pursuing the development of precision genetic medicine at the forefront of biotechnology for rare diseases: gene therapy, RNA-targeted exon skipping, and gene editing. And we’re constantly looking for new ways to tackle rare genetic diseases, which include developing drugs faster with more predictability, differentiated ...
Meet Our Leadership | Sarepta Therapeutics
Learn about Sarepta's leadership and their goal to forever change the course of genetic disease.
Our Disease Areas - Sarepta Therapeutics
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.
Sarepta Therapeutics Announces Expanded US FDA Approval of …
Jun 20, 2024 · As part of a collaboration agreement signed in 2019, Sarepta is working with Roche to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche ...
Pipeline & Products | Sarepta Therapeutics
Sarepta is at the forefront of precision genetic medicine, with over 40 therapies in various stages of development. Our disease areas include Duchenne muscular dystrophy (DMD), six Limb-girdle muscular dystrophies (LGMD), Charcot-Marie-Tooth (CMT), and CNS-related disorders.
Community Letter: Update on LGMD programs SRP-9003, SRP …
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.
- Some results have been removed