Defeat Duchenne Canada is pleased to see REGENXBIO moving its phase 1/2 clinical trial of RGX-202 (AFFINITY DUCHENNE; NCT05693142) into Canadian centres in the first half of 2025.
This is based on completion of the last dose level in the expansion cohort patients ages 4 – 11 years and the first being dosed in the ages 1 – 3 years cohort. REGENXBIO plans to share more information about their research this month, including pivotal trial design and plans for accelerated approval, as well as initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial.
“The rapid progress we are making in RGX-202, which continues to demonstrate its potential to be a best-in-class gene therapy for Duchenne, is highly encouraging.”
Curran M. Simpson, President and Chief Executive Officer of REGENXBIO
RGX-202 is a gene therapy that uses an adeno-associated viral (AAV; a virus that does not cause disease and acts like a package for the healthy gene) vector to deliver a transgene (a manufactured version of a piece of DNA) to deliver a novel microdystrophin which is also in naturally-occurring dystrophin. RGX-202 is a one-time treatment designed to specifically address underlying cause of Duchenne muscular dystrophy, allowing a person with DMD to produce microdystrophin, and prevent damage to skeletal and heart muscles.
Although Sarepta’s ELEVIDYS®is approved for the treatment of ambulatory pediatric patients who are 4 – 5 years, there are no gene therapies available for patients with Duchenne who are under 4 years old have no access to gene therapy. RGX-202 is currently the only gene therapy sponsor recruiting patients in this age group in the U.S. There are no DMD-specific therapies approved in Canada for any age.
For any families who would like more information about Regenxbio’s Canadian trial, please reach out to [email protected].
See the full press release below.
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