Partner Spotlight: 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.
We are ushering in a new era of drug development with the goal of driving efficiencies, including shortening the time from lab to patient and building the world’s largest gene therapy manufacturing capacity. We are collaborating with health networks and payers, rethinking pricing models for revolutionary new treatments in development.
Sarepta holds leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs), and currently has more than 40 programs in various stages of development. Our pipeline is driven by our multi-platform approach across RNA-targeted, gene transfer, and gene-editing platforms.
We are committed to research and education on gene transfer therapy, which aims to slow or stabilize the disease by transferring a new gene into the target cells. Gene transfer research is promising for specific types of conditions, including neuromuscular diseases like DMD.
Click on the link below for a simple overview of “Micro-Dystrophin Gene Therapy Research for Duchenne Muscular Dystrophy (DMD).”
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