Gene therapy for Duchenne is centered on the goal of successfully introducing a smaller, but efficient version of dystrophin into the muscle cell. RGX-202 is an investigational gene therapy product, utilizing a novel adeno-associated virus (AAV8) to transport a shortened version of the dystrophin gene (micro-dystrophin) that may provide benefit in place of the missing full-length dystrophin protein.
A Phase 1/2 study is currently recruiting.
This program is sponsored by REGENXBIO.
ACTIVELY RECRUITING | AFFINITY DUCHENNE - RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) |
|---|
JUNE 2021 | REGENXBIO at the PPMD 2021 Virtual Annual ConferencePre-recorded content for PPMD's 2021 Virtual Annual Conference On-Demand Library |
