REGENXBIO (RGNX) highlighted progress and upcoming anticipated milestones across its pipeline of AAV gene therapies for rare and retinal diseases. Highlights: New Phase I/II RGX-202 functional data demonstrates long-term, durable treatment effect at dose at 18 months; Patient enrollment in confirmatory trial continues, expect majority of patients to be dosed by planned BLA filing, mid-year; Expecting 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 readiness
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