Rockville, Md. – September 24, 2026 -- REGENXBIO Inc. (Nasdaq: RGNX) reported that 60% of Dose Level 3 participants with three-year follow-up data in its Phase II ALTITUDE study achieved a greater-than-2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) after a single in-office treatment with investigational gene therapy sura-vec, with no vision-threatening events recorded among responders.
Three-year data confirm durable DRSS improvement without repeat injections
In long-term follow-up as of August 17, 2026, six of ten Dose Level 3 participants with non-proliferative diabetic retinopathy (NPDR) who reached the three-year visit sustained a greater-than-2-step DRSS improvement without additional diabetic retinopathy treatment. The suprachoroidal delivery used Dose Level 3 (1.0x10^12 GC/eye) combined with short-course prophylactic topical steroids.
Three of four participants who showed a 1-step DRSS improvement at one year without supplemental anti-VEGF injections progressed to a greater-than-2-step improvement by year three, also without additional treatment.
No new safety signals or intraocular inflammation emerge through three years
Across 17 participants followed at three years, REGENXBIO recorded no new sura-vec-related safety signals and no cases of intraocular inflammation (IOI) under the short-course steroid regimen. The results align with previously disclosed 2.5-year Dose Level 3 data from the same trial.
REGENXBIO advances Dose Level 3 into pivotal NAAVIGATE trial
Dose Level 3 is now being evaluated in the Phase IIb/III NAAVIGATE trial for NPDR, developed in collaboration with AbbVie. Sura-vec (surabgene lomparvovec, ABBV-RGX-314) uses REGENXBIO's NAV AAV8 vector to encode a VEGF-inhibiting antibody fragment, targeting a disease that affects nearly 10 million people in the United States and remains the leading cause of vision loss among adults aged 24 to 75 worldwide.
"These three-year ALTITUDE results are encouraging and support continued study of sura-vec as a one-time, in-office treatment with the potential to change the course of the disease," said Nikolas London, M.D., an ALTITUDE study investigator at Retina Consultants San Diego.
Current NPDR management relies on "watchful waiting" or repeat anti-VEGF injections, a treatment burden REGENXBIO says its one-time approach could reduce or eliminate. The data were presented at the Retina Society 59th Annual Scientific Meeting in Los Angeles.