Early Wins: RegenXBio's Gene Therapy Helps Duchenne Patients Walk Stronger, Longer

Core Insights - RegenXBio Inc. has released new interim data from the Phase 1/2 AFFINITY DUCHENNE trial, showcasing positive functional, safety, and biomarker data for RGX-202, a gene therapy for Duchenne muscular dystrophy [1][2]. Group 1: Trial Data and Results - The trial involved five patients aged six to twelve years who received RGX-202, demonstrating consistent benefits in functional data among dose level 2 participants at 9 and 12 months post-treatment [1]. - At 9 months, RGX-202 participants showed improved function, exceeding external controls on all measures, with an average improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from baseline and 4.8 points compared to natural history [3]. - At 12 months, RGX-202 participants continued to demonstrate improved performance on timed function tests and NSAA, with an average improvement of 4.5 points from baseline and 6.8 points compared to natural history [4]. Group 2: Future Plans and Market Context - RegenXBio plans to share topline data for RGX-202 in the first half of 2026 and aims to submit a Biologics License Application (BLA) under the accelerated approval pathway by mid-2026, with commercial readiness activities underway for a potential launch in 2027 [5]. - The competitive landscape includes Sarepta Therapeutics, which recently reported a patient death following treatment with its approved gene therapy Elevidys, leading to a therapeutic clinical hold on several studies in the EU [6].