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
Building an industry-leading genetic medicine pipeline. 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 …
News & Press Releases - Sarepta Therapeutics
Read news and articles from Sarepta, a global biotechnology company developing potentially life-changing precision genetic medicine.
Meet Our Leadership | Sarepta Therapeutics
Learn about Sarepta's leadership and their goal to forever change the course of genetic disease.
Sarepta Therapeutics Announces Topline Results from EMBARK, a …
Oct 30, 2023 · Sarepta Therapeutics Announces Topline Results from EMBARK, a Global Pivotal Study of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy. – Results support submission of an efficacy supplement to the BLA; US FDA has indicated openness to reviewing the data for label expansion based on the totality of evidence from EMBARK.
Investor Relations | Sarepta Therapeutics, Inc.
The Investor Relations website contains information about Sarepta Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
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.
Sarepta Therapeutics Announces That U.S. FDA has Accepted for …
Nov 28, 2022 · Sarepta Therapeutics Announces That U.S. FDA has Accepted for Filing and Granted Priority Review for the Biologics License Application for SRP-9001, Sarepta’s Gene Therapy for the Treatment of Ambulant Individuals with Duchenne Muscular Dystrophy. – Regulatory action date of May 29, 2023.
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.
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