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.
RGX-202 is currently in pre-clinical development with plans to submit an IND in mid-2021 to enable clinical trials.
This program is sponsored by REGENXBIO.
REGENXBIO at the PPMD 2021 Virtual Annual Conference
Pre-recorded content for PPMD's 2021 Virtual Annual Conference On-Demand Library