Gene therapy is coming of age. Sarepta’s gene therapy engine provides a framework for potentially creating new therapies for these devastating diseases.

Sarepta’s gene therapy engine provides a framework for creating a steady stream of new therapies for devastating diseases, such as limb-girdle muscular dystrophies.

Jun 22, 2023 · Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene Therapy to Treat Duchenne Muscular Dystrophy

Apr 8, 2025 · See our pipeline for RNA technologies, gene therapy, and gene editing to learn about our research in rare diseases.

Our programs and research focus span several therapeutic modalities, including RNA-targeted exon-skipping, gene therapy and gene editing. Learn more.

To create the micro-dystrophin transgene, scientists at Sarepta researched and carefully selected the parts of the dystrophin gene to use in micro-dystrophin. These sections were combined to …

May 13, 2025 · 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 …

How a collaboration in the lab led to a seat at the table in Sarepta’s Gene Editing Innovation Center

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double …

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