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

Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...

REGENXBIO Inc. (Nasdaq: RGNX) today announced that AFFINITY DUCHENNE®, the multi-center, open-label trial of RGX-202, a ...

Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...

Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. | Regenxbio’s investigational gene therapy improved muscle ...

a potential best-in-class gene therapy for Duchenne muscular dystrophy, has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety data ...

A small trial of an experimental vaccine has shown promising results for patients with the most aggressive type of breast ...

Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with ...

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

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...