REGENXBIO(US:RGNX) Prnewswire·2026-01-11 20:00Core Insights - REGENXBIO Inc. is poised for a transformative year in 2026, entering the commercial stage with two near-term catalysts from its late-stage assets and a clear path to sustained growth [2] - The company has reported positive long-term data for its Duchenne program, indicating effective therapeutic benefits across its gene therapy pipeline [2] Clinical Program Updates and 2026 Anticipated Milestones - New functional data from the Phase I/II AFFINITY DUCHENNE trial for RGX-202 shows that all four patients exceeded expected disease trajectory, improving an average of 7.4 points on the North Star Ambulatory Assessment (NSAA) [3] - The company plans to present additional safety, biomarker, and functional data at the MDA Clinical and Scientific Conference in March 2026 [3] - REGENXBIO expects to submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026, following the completion of enrollment in the pivotal trial [7] Regulatory and Commercial Readiness - The FDA PDUFA target date for the Duchenne program is February 8, 2026, with potential approval leading to a Priority Review Voucher (PRV) [7] - REGENXBIO is collaborating with Nippon Shinyaku for the commercialization of clemidsogene lanparvovec (RGX-121) upon potential approval [7] - The company is enhancing its manufacturing capabilities at its Manufacturing Innovation Center in Rockville, Maryland, to support commercial launches [8] Gene Therapy Advancements - REGENXBIO is advancing its AAV gene delivery technology through capsid discovery and engineering, approaching IND readiness for treating geographic atrophy [9] - The company is on track to make surabgene lomparvovec (sura-vec) the first gene therapy for wet age-related macular degeneration (AMD) [12] - A two-part Phase IIb/III trial for sura-vec in diabetic retinopathy is set to begin, with a $100 million milestone payment from AbbVie expected upon the first patient dosing [12]