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REGENXBIO Highlights Key 2026 Catalysts and Announces Positive Long-Term Functional Outcomes in Lead Duchenne Gene Therapy Program
New Phase I/II RGX-202 functional data demonstrates long-term, durable treatment effect at pivotal dose at 18 months Robust patient enrollment in confirmatory
About this update from Regenxbio Inc.
[{"type":"text","content":"New Phase I/II RGX-202 functional data demonstrates long-term, durable treatment effect at pivotal dose at 18 months Robust patient enrollment in confirmatory trial continues, expect majority of patients to be dosed by planned BLA filing, mid-yearExpecting FDA PDUFA decision and multiple pivotal top-line data readouts in 2026 to support potential commercial launches 2026-2028 In-house manufacturing and strategic global partnerships driving commercial readinessPresentation at 44th Annual J.P. Morgan Healthcare Conference Wednesday, January 14ROCKVILLE, Md., Jan. 11, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) highlighted progress and upcoming anticipated milestones across its pipeline of AAV gene therapies for rare and retinal diseases. \"2026 is set to be a transformative year for REGENXBIO, as we enter commercial stage with two near-term catalysts from our three late-stage assets and a clear path to sustained growth,\" said Curran Simpson, President and CEO, REGENXBIO. \"We are starting the year with exciting new long-term data for our Duchenne program, demonstrating how our comprehensive strategy to maximize the potential for therapeutic benefit across all our programs is resulting in positive outcomes for patients. We are continuing to set the bar high for how potentially life-changing gene therapies are discovered, developed, and manufactured; this year we are sharply focused on advancing our commercial readiness to enable successful launches of these medicines for patients in need.CLINICAL PROGRAM UPDATES AND 2026 ANTICIPATED MILESTONES RGX-202 for Duchenne Muscular DystrophyNew Functional DataREGENXBIO today announced new, 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). All patients exceeded expected disease trajectory on the North Star Ambulatory Assessment (NSAA) using the established cTAP disease progression model. RGX-202 recipients improved an average of 7.4 points compared to cTAP. These same patients improved an average of 6.6 points compared to cTAP at 12 months post-treatment. The Company plans to share additional Phase I/II safety, biomarker, and functional data at the MDA Clinical and Scientific Conference in March 2026.Clinical Trial and Regulatory Milestones REGENXBIO expects to share pivotal topline data in e...