This clinical trial update was written by Defeat Duchenne Canada Research Volunteer, Omar Sheikh
The AFFINITY DUCHENNE clinical trial for the RGX-202 gene therapy is arriving at centers across Canada. Three centers have been activated (open for enrollment) in Canada now, with two more expected before the end of the year.
These activated centers are:
- Dr. Kathryn Selby – Vancouver, BC; BC Children’s Hospital
- Dr. Craig Campbell – London, ON; Children’s Hospital, London Health Science Centre
- Dr. Hugh McMillan – Ottawa, ON; Children’s Hospital of Eastern Ontario
The centers that should enroll soon are:
- Dr. Jean Mah – Calgary, AB; Alberta Children’s Hospital
- Dr. Hernan Gonorazky – Toronto, ON; The Hospital for Sick Children
RGX-202 is a gene therapy made by REGENXBIO that produces a smaller form of dystrophin to treat DMD. In June 2025, REGENXBIO revealed results from the Phase I/II AFFINITY DUCHENNE trial of RGX-202’s effectiveness and safety.
The gene therapy did produce measurable dystrophin protein in treated patients, but it must be verified through further study. Treatment was well-tolerated with no hospitalization-requiring (serious adverse) events, including no drug-induced liver injury, myocarditis, or myositis. Some common drug-related events were nausea, vomiting, and fatigue – an expected side effect of gene therapy.
This trial is expected to support a Biologics License Application (BLA) submission at the end of 2025 to the U.S. FDA.
Defeat Duchenne Canada is proactively following up with REGENXBIO for details relevant to Canadian patients.