REGENXBIO's sura-vec holds vision gains through 5 years in a 42-patient wet AMD cohort
Cohorts 3 and 4 of the Phase 1/2a trial showed stable-to-improved vision and fewer anti-VEGF injections at five years, with the pivotal readout still ahead in Q4 2026.
Executive Summary
- REGENXBIO reported five-year follow-up data from a completed Phase 1/2 cohort of its gene therapy sura-vec in wet age-related macular degeneration, showing durable vision and fewer anti-VEGF injections in patients who had needed frequent treatment before receiving the one-time therapy.
- The result extends a safety and durability signal already observed in earlier follow-up from this open-label cohort; it does not introduce a new randomized comparison and carries the same lack of a concurrent control arm the trial was designed with.
- The dosing regimen in this cohort matches what is being tested in two ongoing randomized, active-controlled pivotal trials against approved anti-VEGF therapies, due to read out in the fourth quarter of 2026.
- Sura-vec is one of several gene therapy programs pursuing one-time treatment of wet AMD, an indication where no gene therapy has yet reached approval and anti-VEGF injections given every four to eight weeks remain the standard of care.
The disclosure
REGENXBIO Inc. presented long-term follow-up data from Cohorts 3 and 4 of its Phase 1/2a trial of surabgene lomparvovec (sura-vec, also known as ABBV-RGX-314) at the American Society of Retina Specialists' 44th Annual Meeting in Montreal on July 18, 2026. The trial, registered as NCT03066258, enrolled 42 patients with neovascular (wet) AMD who required prior anti-VEGF injections, and completed in June 2021. Participants in Cohorts 3 and 4 received subretinal sura-vec at doses similar to those used in the ongoing ATMOSPHERE and ASCENT pivotal trials. REGENXBIO+1REGENXBIO Presents Positive Long-Term Data for Surabgene Lomparvovec in Wet AMD and Diabetic Retinopathy at American Society of Retina Specialists Annual MeetingJul 18, 2026Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD TrialNCT03066258
The result
At five years, patients showed stable-to-improved best-corrected visual acuity and reductions in anti-VEGF treatment burden, apart from one polypoidal choroidal vasculopathy participant in Cohort 4 who remained refractory to anti-VEGF therapy, the company said. No new safety signals emerged in long-term follow-up, including no intraocular inflammation among participants who completed the study, in a setting with no prophylactic steroids. Steve Pakola, REGENXBIO's chief medical officer, said the data show sura-vec's potential "to preserve vision long-term and change the treatment paradigm for patients with chronic retinal disease". The trial's own registered safety endpoint tracked ocular and non-ocular adverse events through 26 weeks; the five-year update is a secondary, longer-horizon follow-on to that original safety window. REGENXBIO+1REGENXBIO Presents Positive Long-Term Data for Surabgene Lomparvovec in Wet AMD and Diabetic Retinopathy at American Society of Retina Specialists Annual MeetingJul 18, 2026Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD TrialNCT03066258
How it was done
The trial was an open-label (unblinded), non-randomized, five-arm dose-escalation study with no concurrent control group, evaluating five ascending doses of sura-vec by subretinal injection. Cohorts 3 and 4 received doses of 6E10 and 1.6E11 genome copies per eye, respectively, the doses closest to those used in the ongoing pivotal program. The primary completion date was November 2019, with the trial fully completing in June 2021; REGENXBIO also disclosed a comparison of one-year outcomes for sura-vec against a real-world, matched external control cohort of anti-VEGF-treated patients at the same meeting. NCT03066258+1Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD TrialNCT03066258REGENXBIO Presents Positive Long-Term Data for Surabgene Lomparvovec in Wet AMD and Diabetic Retinopathy at American Society of Retina Specialists Annual MeetingJul 18, 2026
What's ahead
REGENXBIO said it expects to report topline data with AbbVie from the ATMOSPHERE and ASCENT pivotal trials in the fourth quarter of 2026. Those trials are randomized, active-controlled studies testing sura-vec against ranibizumab and aflibercept, respectively, with a primary endpoint of non-inferiority in BCVA change from baseline at 54 weeks and one year. Together the two pivotal trials have enrolled more than 1,200 patients across more than 200 sites, a scale that will let the non-inferiority comparison run with a concurrent control arm this early cohort never had. REGENXBIOREGENXBIO Presents Positive Long-Term Data for Surabgene Lomparvovec in Wet AMD and Diabetic Retinopathy at American Society of Retina Specialists Annual MeetingJul 18, 2026
The competitive frame
Gene therapy is a modality actively pursued in wet AMD: 21 trials on record have used gene therapy in this indication, including AbbVie's own follow-on sura-vec pivotal trial (NCT07007065), 4D Molecular Therapeutics' 4D-150 (NCT05197270), and Adverum Biotechnologies' ixoberogene soroparvovec (NCT07630649). None of these gene therapy candidates has reached approval in wet AMD; approved options in the indication remain intravitreal biologics such as faricimab, ranibizumab, and aflibercept, all requiring recurring injections. Against that backdrop, the bar this cohort's five-year durability data sets for the pivotal readout is straightforward: showing the same anti-VEGF-injection-sparing effect holds up against an active comparator, in a randomized design, at the trial's prespecified non-inferiority margin, rather than in an open-label cohort with no concurrent control.
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