Regenxbio reported interim data from the Phase I/II AFFINITY DUCHENNE trial evaluating RGX-202 for Duchenne muscular dystrophy, and the results were presented by Carolina Tesi-Rocha at the MDA Clinical and Scientific Conference. In seven participants treated at 2x10^14 GC/kg, NSAA improved by 5 points versus the cTAP model at 1 year. Cardiac MRI measures in these seven participants showed left ventricular ejection fraction of 62% at baseline and 62% at 12 months. In biomarker analyses (n=13), an additional patient had microdystrophin expression of 51% at Week 12, and the pivotal phase primary endpoint is the proportion of participants with expression above 10% at Week 12. Regenxbio said pivotal topline data are expected in early Q2 2026, with a pre-BLA meeting request planned for mid-2026.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Regenxbio Inc. published the original content used to generate this news brief via PR Newswire (Ref. ID: 202603111145PR_NEWS_USPR_____PH07897) on March 11, 2026, and is solely responsible for the information contained therein.