Business Description
Sarepta Therapeutics is a Massachusetts-based biotech company focused on developing treatments for rare genetic diseases, particularly Duchenne muscular dystrophy. The company has brought several approved therapies to market that target specific genetic mutations causing Duchenne, along with a gene therapy called ELEVIDYS. Beyond its current products, Sarepta has a pipeline of experimental treatments for other muscle-wasting conditions, using gene therapy and RNA-silencing technologies. The company works alongside leading research institutions and pharmaceutical partners to advance its science.
