Business
REGENXBIO Reports Fourth Quarter and Full Year 2025 Financial Results and Operational Highlights
Late-stage gene therapy pipeline for rare and retinal diseases advancing toward key catalysts RGX-202 for Duchenne muscular dystrophy: New Phase I/II data at
About this update from Regenxbio Inc.
[{"type":"text","content":"Late-stage gene therapy pipeline for rare and retinal diseases advancing toward key catalysts RGX-202 for Duchenne muscular dystrophy: New Phase I/II data at MDA, topline pivotal results expected early Q2 2026Robust patient enrollment continues in confirmatory trialSurabgene lomparvovec (sura-vec, ABBV-RGX-314) subretinal wet AMD topline pivotal data expected in Q4 2026; first patient dosed in pivotal Phase IIb/III trial in diabetic retinopathy expected in Q2 2026Company to engage FDA on potential path forward for RGX-121Conference call today at 8:00 a.m. ETROCKVILLE, Md., March 5, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and operational highlights for the fourth quarter and year ended December 31, 2025.\"We are rapidly advancing our late-stage pipeline of gene therapies to treat rare and retinal diseases with significant unmet need, with multiple near-term catalysts in 2026,\" said Curran Simpson, President and Chief Executive Officer, REGENXBIO. \"We will drive continued momentum across our programs – powered by our fully in-house, end-to-end capabilities, commercial-ready manufacturing, and global partners. We remain focused on executing on our mission to deliver meaningful new medicines to patients in need through the curative potential of gene therapy.\"PROGRAM HIGHLIGHTS AND MILESTONESNeuromuscular Disease: RGX-202 is a potential best-in-class gene therapy for Duchenne muscular dystrophy (Duchenne). RGX-202 is designed to address the underlying cause of Duchenne by enabling targeted expression of a novel microdystrophin that is closest to naturally occurring dystrophin. It is the only microdystrophin that includes the C-Terminal domain, which has been shown to protect and preserve muscle function. The differentiated therapeutic approach behind RGX-202 includes a novel construct, a proactive immune suppression regimen, and a suspension-based manufacturing process that delivers industry-leading product purity levels. RGX-202 is designed for improved muscle function, durability and safety outcomes for patients.Positive 18-month functional data from patients treated with the pivotal dose in the Phase I/II portion of the AFFINITY DUCHENNE® trial (n=4) were reported in January 2026. All patients exceeded expected disease trajectory on the North Star Ambulatory Assessment (NSAA) using th...