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

The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne gene therapies despite questions about their effectiveness.

Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often too large to fit inside the viral vector used for delivery. | Gene therapy for some diseases,

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy for Duchenne muscular dystrophy (DMD), following positive data from the Phase I/II portion of its AFINITY DUCHENNE trial.