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

AFFINITY DUCHENNE a Phase 1/2 study is not yet recruiting. The FDA approved the IND for RGX-202 and it is anticipated to begin enrollment in the first half of 2022.

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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