In this condition, patients' bodies don't make enough for the dystrophin protein necessary to keep muscles intact. Sarepta ...

Children born with the degenerative disease have a genetic mutation that stops them producing dystrophin, a protein that is vital for muscle strength and function. The same disorder also occurs in ...

The company made its debut last year with a $51 million Series A and a licensing agreement with Astellas for a gene therapy ...

Regenxbio hopes to use the study's results to ask the U.S. Food and Drug Administration to grant accelerated approval to the ...

StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.

Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy ...