Regenxbio shares long-term gene therapy data for eye diseases

Regenxbio Inc. (Nasdaq: RGNX) presented long-term follow-up data for its investigational gene therapy surabgene lomparvovec (sura-vec) in wet age-related macular degeneration (AMD) and diabetic retinopathy (DR) at the American Society of Retina Specialists 44th Annual Meeting in Montreal, Canada.

In wet AMD, participants in Cohorts 3 and 4 of a Phase I/IIa trial who received a single subretinal administration of sura-vec at doses similar to those used in the ongoing ATMOSPHERE and ASCENT pivotal trials showed stable to improved visual acuity and reduced anti-VEGF injection burden through five years. No intraocular inflammation was observed in long-term follow-up. A separate external control study showed that sura-vec participants maintained or improved best-corrected visual acuity gains and received fewer injections compared to a matched real-world anti-VEGF control group at one year.

In diabetic retinopathy, data from the Phase II ALTITUDE trial at 2.5 years showed that 55% of non-proliferative DR participants receiving sura-vec via suprachoroidal delivery at Dose Level 3 achieved a two-step or greater improvement on the Diabetic Retinopathy Severity Scale without additional treatment. Seventy percent experienced no vision-threatening events. No intraocular inflammation was observed through 2.5 years in 17 participants using short-course prophylactic topical steroids.

Regenxbio said it expects to report topline data from the ATMOSPHERE and ASCENT pivotal trials, conducted in collaboration with AbbVie, in the fourth quarter of 2026. Those trials have enrolled more than 1,200 participants across more than 200 sites and are evaluating sura-vec against ranibizumab and aflibercept, respectively, with a primary endpoint of non-inferiority in vision change at 54 weeks and one year.

Sura-vec uses an AAV8 vector to deliver a gene encoding an antibody fragment intended to inhibit vascular endothelial growth factor, which is associated with abnormal blood vessel growth in the retina.

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