Positive efficacy and safety data in hand, Regenxbio has advanced its gene therapy for Duchenne muscular dystrophy to pivotal stage and dosed its first patient.
RGX-202 is designed to support the delivery and targeted expression of genes throughout skeletal and heart muscle using the proprietary NAV AAV8 vector and a well-characterized muscle-specific promoter (Spc5-12).
In the phase 1/2 portion of the ongoing AFFINITY DUCHENNE trial, all five participants who received RGX-202 across both dose levels experienced a positive impact on disease trajectory, with patients demonstrating stable or improved function on the North Star Ambulatory Assessment and timed function test.
Regenxbio also announced new biomarker data that continues to support consistent, high expression and transduction of RGX-202 microdystrophin. RGX-202 was appropriately localized to the sarcolemma, demonstrating the differentiated construct with the CT-Domain is appropriately targeting the muscle.
Now, based on the strength of this phase 1/2 data and positive discussions and alignment with the FDA, the trial has been expanded into a multicenter, open-label pivotal phase 1/2/3 trial of RGX-202, which is expected to support a BLA submission using the accelerated approval pathway in 2026.
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