REGENXBIO reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, a differentiated investigational gene therapy for Duchenne muscular dystrophy (Duchenne). Results were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.
Key findings from Rgx-202 include:
- New biomarker data from two patients who received the pivotal dose of RGX-202 were presented at MDA and continue to support consistent, robust expression and transduction of RGX-202 microdystrophin across all ages.
- In a patient aged 3 at dosing, microdystrophin expression was measured to be 122.3% compared to control. Patients under 4 years old have no access to gene therapy, and REGENXBIO is the only gene therapy sponsor recruiting patients in this age group in the U.S.
- In a patient aged 7 years old, RGX-202 microdystrophin expression was measured to be 31.5% compared to control.
- In all patients, RGX-202 was appropriately localized to the sarcolemma, demonstrating the differentiated construct with the CT-Domain is appropriately targeting the muscle.
- RGX-202 microdystrophin expression results in ambulatory patients aged 8+ are the highest reported microdystrophin levels across approved or investigational gene therapies.
- To support a Biologics License Application (BLA) using the accelerated approval pathway, the primary endpoint in the pivotal phase of AFFINITY DUCHENNE is the proportion of participants whose RGX-202 microdystrophin expression is ≥10% at Week 12.
- RGX-202 also continues to demonstrate the highest reported vector genome copies (4.9-55.4) measured by qPCR across approved or investigational gene therapies.
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