REGENXBIO presents three-year durability data for gene therapy in diabetic retinopathy
REGENXBIO announced positive three-year follow-up data for its investigational gene therapy, surabegene lomparvovec, in diabetic retinopathy. The treatment demonstrated durable efficacy without vision-threatening events.
ROCKVILLE, Maryland – REGENXBIO Inc. announced on September 24, 2026, positive long-term follow-up data from its Phase II ALTITUDE study for the investigational gene therapy surabegene lomparvovec (sura-vec) in treating diabetic retinopathy (DR). The data, presented at the Retina Society's 59th Annual Scientific Meeting, showed sustained efficacy and safety.
The study results indicated that a majority of participants in Dose Level 3 with non-proliferative diabetic retinopathy (NPDR) achieved a greater than two-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) three years after a single administration of sura-vec via suprachoroidal delivery. These participants experienced no vision-threatening events and required no additional DR treatment. Short-course prophylactic topical steroids were used without observed intraocular inflammation.
"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. "The three-year data continue to show meaningful improvements in DRSS alongside a reduction in disease progression."
Diabetic retinopathy is a leading cause of vision loss among adults globally and affects nearly 10 million people in the United States. Current treatments often require repeat injections, posing a significant burden. A one-time administration of sura-vec could potentially reduce or eliminate the need for ongoing intraocular injections and lower the risk of vision-threatening complications.
REGENXBIO is developing sura-vec for a range of chronic retinal diseases. The company focuses on advancing one-time treatments for rare and retinal diseases using its AAV gene therapy technology.