Regenxbio doses first patient in diabetic retinopathy gene therapy trial

Regenxbio Inc. (NASDAQ: RGNX) announced that the first patient has been dosed in the Phase IIb/III NAAVIGATE clinical trial of investigational surabgene lomparvovec in diabetic retinopathy, using suprachoroidal delivery. The company said it will receive $100 million from AbbVie as a result of this milestone.

The NAAVIGATE trial is a multicenter, randomized, masked, sham-controlled study evaluating the safety and efficacy of the therapy in patients with non-proliferative diabetic retinopathy without center-involved diabetic macular edema. The primary endpoint is a two-step or greater improvement on the diabetic retinopathy severity scale at one year. The Phase IIb portion, operationalized by Regenxbio, is expected to enroll approximately 135 participants in the United States.

Surabgene lomparvovec, also known as sura-vec or ABBV-RGX-314, is an investigational gene therapy that uses the NAV AAV8 vector to encode an antibody fragment designed to inhibit vascular endothelial growth factor. The therapy is being developed in collaboration with AbbVie.

In two-year data from dose level 3 of the Phase II ALTITUDE trial, sura-vec showed no intraocular inflammation in 15 patients. Regenxbio plans to present two-and-a-half-year data from the ALTITUDE long-term follow-up study at the American Society of Retina Specialists 44th Annual Meeting in July 2026. Five-year data from a Phase I/IIa study of subretinal sura-vec for wet age-related macular degeneration will also be presented at the same meeting.

Regenxbio expects to announce topline data with AbbVie from the ATMOSPHERE and ASCENT pivotal trials of sura-vec using subretinal delivery in the fourth quarter of 2026.

Diabetic retinopathy affects nearly 10 million people in the United States and is a leading cause of vision loss in adults between 24 and 75 years of age worldwide, according to figures cited in the company's press release.

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