Majority of Dose Level 3 participants with NPDR demonstrated a > 2-step DRSS improvement at 3 years post-one-time, in-office sura-vec gene therapy treatment, with no vision-threatening events and no additional treatment for diabetic retinopathy
No IOI observed with short-course prophylactic topical steroids
ROCKVILLE, Md., Sept. 24, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced positive long-term follow-up data from the Phase II ALTITUDE® study of investigational surabgene lomparvovec (sura-vec, ABBV-RGX-314) in diabetic retinopathy (DR) using suprachoroidal delivery. These new data were presented at the Retina Society 59th Annual Scientific Meeting in Los Angeles.
"These latest long-term results are highly encouraging and reinforce the potential of sura-vec to change the treatment landscape for the millions of patients with non-proliferative diabetic retinopathy," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "Building on the positive data seen at two and two and a half years after a single treatment, the three-year data continues to show meaningful improvements in DRSS alongside a reduction in disease progression. The durability of these results strengthens our conviction in sura-vec as a potentially transformative, one-time treatment."
"For patients with non-proliferative diabetic retinopathy, the goal is to intervene before the disease progresses to vision-threatening complications and irreversible vision loss. However, current preventive treatment requires repeat injections, which is a significant burden for a population that is largely working-age adults," said Nikolas London, M.D., FACS, FASRS, Retina Consultants San Diego, ALTITUDE study investigator. "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. A single treatment that reduces or eliminates the need for ongoing intraocular injections, and reduces the risk of vision-threatening complications, would be a meaningful change for our diabetic patients."
Phase II ALTITUDE Long-term follow up data (as of August 17, 2026)
In long-term follow-up from the Phase II ALTITUDE® trial, a single in-office administration of sura-vec using suprachoroidal delivery in participants with non-proliferative DR (NPDR) at Dose Level 3 (1.0×10¹² GC/eye) with short-course prophylactic topical steroids demonstrated a durable efficacy profile at three years with no new sura-vec-related safety signals observed during long-term follow up. 60% of all Dose Level 3 participants with three-year visits (n=6/10) achieved >2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) without additional treatment for diabetic retinopathy.1 These participants did not experience any vision-threatening events.