Defeat Duchenne Canada is pleased to see the updated positive safety and effectiveness results REGENXBIO Inc. announced this morning from the Phase 1/2/3 AFFINITY DUCHENNE® study of RGX-202, 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.
In the current study:
- RGX-202 demonstrated consistent evidence of positively changing disease trajectory for Duchenne at all dose levels; two participants exceeded external unaffected people used as comparison on all functional measures
- Biomarker (a characteristic of a disease that is measurable, such as dystrophin levels for people with Duchenne) data demonstrate consistent, robust microdystrophin expression and transduction levels across all treated ages; one new participant aged 2 years at dosing had expression level at 118.6% compared to unaffected people used for comparison
- Favorable safety profile continues with no serious adverse events or adverse events of special interest observed
A community letter is expected later today. In the meantime, please see the full press release below.
Defeat Duchenne Canada will also be hosting an educational webinar featuring Dr. Jahannaz Dastgir, Executive Director of Clinical Development at REGENXBIO, as she shares the latest updates on RGX-202. Register for free below.
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: