With pivotal Phase III ATMOSPHERE and ASCENT trials set to report topline data in Q4 2026, Regenxbio presented the first long-term follow-up from dose cohorts matching those studies, showing stable to improved vision through five years after a single administration of surabgene lomparvovec (sura-vec, ABBV-RGX-314). Regenxbio (Nasdaq: RGNX) reported that patients in Cohorts 3 and 4 of a Phase I/IIa trial maintained stable to improved vision through five years after a single subretinal dose of surabgene lomparvovec (sura-vec, ABBV-RGX-314), with no drug-related intraocular inflammation despite the absence of prophylactic steroids. The data provide the longest clinical look yet at the durability of the gene therapy ahead of its registrational readouts.
Developed in partnership with AbbVie since 2021, sura-vec uses an AAV8 vector to deliver a gene encoding an anti-VEGF antibody fragment, enabling sustained intraocular VEGF suppression after a single treatment rather than repeated intravitreal injections.
The five-year cohort is notable because participants had documented histories of requiring frequent anti-VEGF injections before receiving sura-vec, a harder-to-treat population than the treatment-naive patients typically enrolled in comparable durability studies. A separate external control analysis, also presented at ASRS, found that sura-vec recipients preserved vision better than real-world anti-VEGF-treated patients at one year, with fewer injections and less fluctuation in central retinal thickness. Sura-vec has no existing regulatory approval; it remains investigational pending the ATMOSPHERE and ASCENT readouts, which will compare it head-to-head against ranibizumab and aflibercept, respectively, on non-inferiority in visual acuity.
Regenxbio also disclosed 2.5-year data from the Phase II ALTITUDE trial testing sura-vec via suprachoroidal injection in diabetic retinopathy, using the same dose level now advancing in the Phase IIb/III NAAVIGATE trial. Among 17 evaluable patients with non-proliferative disease, 55% achieved at least a two-step improvement on the Diabetic Retinopathy Severity Scale without additional treatment, and 70% avoided vision-threatening events; this cohort received short-course prophylactic topical steroids, and no intraocular inflammation was observed. The suprachoroidal route allows in-office delivery without surgery, distinguishing it from the subretinal approach used in wet AMD.