RGX-202

RGX-202

Status

Pre-Clinical

Therapeutic Approach

Restoring or Replacing Dystrophin

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.

Status

RGX-202 is currently in pre-clinical development with plans to submit an IND in mid-2021 to enable clinical trials.

Sponsor

This program is sponsored by REGENXBIO.

Media

JUNE 2021

REGENXBIO at the PPMD 2021 Virtual Annual Conference


Pre-recorded content for PPMD's 2021 Virtual Annual Conference On-Demand Library

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