CureDuchenne is delighted to share the positive updates from REGENXBIO on their Phase 1/2 open-label clinical trial of RGX-202, an AAV8-delivered microdystrophin with an extended C-terminal domain. RGX-202 demonstrated robust microdystrophin expression at both dose levels, as well as functional improvements in individuals (aged 4-11) treated compared to natural history controls.
REGENXBIO has expanded the trial into the pivotal phase, and has already dosed the first participant. The pivotal trial, which is open-label, is planning to enroll 30 ambulatory individuals aged 1 year and above. Individuals will be dosed with RGX-202 at a dose of 2 x 1014 genome copies/kg, and there will not be a placebo arm in the study. Data from this trial is expected to support a Biologics License Application (BLA) submission to the FDA using the accelerated approval pathway in 2026.
Thank you to our own Chief Scientific Officer, Mike Kelly, for helping to put REGENXBIO’s findings into context for our community on REGENXBIO’s webcast.
Link to Investor Call: https://wsw.com/webcast/cc/rgnx4/1447506
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