Regenxbio (RGNX) has reported positive long-term follow-up data for surabgene lomparvovec, or sura-vec, showing durable benefits in patients with wet age-related macular degeneration and diabetic retinopathy following a single administration.
The investigational gene therapy was delivered subretinally in patients with wet age-related macular degeneration, or wet AMD, and through the suprachoroidal space in patients with diabetic retinopathy. The findings were presented at the American Society of Retina Specialists’ 44th Annual Meeting in Montreal, Canada.
In the wet AMD study, patients who had required frequent anti-vascular endothelial growth factor injections before receiving sura-vec maintained stable or improved vision for up to five years. Regenxbio said no drug-related intraocular inflammation was observed despite patients not receiving prophylactic steroids.
The diabetic retinopathy data also indicated a sustained treatment effect. At 2.5 years, sura-vec prevented vision-threatening events without the need for additional treatment, while a majority of participants with non-proliferative diabetic retinopathy achieved an improvement of more than two steps on the Diabetic Retinopathy Severity Scale.
No intraocular inflammation was reported in the diabetic retinopathy study, in which participants received a short prophylactic course of topical steroids.
According to Regenxbio, the benefits were observed at doses comparable to those being assessed in its ongoing late-stage studies. The company expects to announce topline results from the pivotal Atmosphere and Ascent trials in wet AMD in the fourth quarter of 2026.
