Regenxbio stock gains on positive gene therapy trial data

September 25, 2026 9:09 AM EDT

Investing.com -- Regenxbio Inc (NASDAQ: RGNX) shares rose 4.8% in premarket trading Friday after the company presented positive three-year durability data for surabgene lomparvovec in diabetic retinopathy at the Retina Society Annual Scientific Meeting.

The Phase II ALTITUDE study showed that 60% of Dose Level 3 participants with three-year visits achieved greater than 2-step improvement on the Diabetic Retinopathy Severity Scale without additional treatment for diabetic retinopathy. These participants did not experience any vision-threatening events.

The data showed that the majority of participants who achieved a 1-step DRSS improvement at one year without supplemental anti-VEGF injections went on to achieve a greater than 2-step DRSS improvement by three years without additional treatment for diabetic retinopathy.

The study involved a single in-office administration of sura-vec using suprachoroidal delivery in participants with non-proliferative diabetic retinopathy at Dose Level 3 with short-course prophylactic topical steroids. No intraocular inflammation was observed through three years with the treatment regimen.

Surabgene lomparvovec is a one-time investigational gene therapy designed to deliver sustained treatment effect in wet age-related macular degeneration, diabetic retinopathy and other chronic retinal conditions. The therapy uses the NAV AAV8 vector to encode an antibody fragment designed to inhibit vascular endothelial growth factor.

Diabetic retinopathy affects nearly 10 million people in the United States and is the leading cause of vision loss in adults between 24 and 75 years of age worldwide.

The stock movement follows Wednesday's announcement that Regenxbio and The Trustees of the University of Pennsylvania reached a $39.0 million settlement agreement with Sarepta Therapeutics to resolve patent infringement litigation related to Sarepta's Duchenne muscular dystrophy gene therapy ELEVIDYS.

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