Rockville, Maryland, U.S., September 24, 2026
REGENXBIO Inc. has presented three-year clinical data for surabgene lomparvovec in patients with diabetic retinopathy, highlighting continued treatment durability in the Phase II ALTITUDE® study. The findings were presented at the Retina Society Annual Scientific Meeting and provide additional follow-up data for the investigational gene therapy being developed as a potential treatment for diabetic retinopathy. Surabgene lomparvovec is an investigational, one-time gene therapy designed to deliver a functional copy of the RGX-314 transgene, which is intended to inhibit vascular endothelial growth factor (VEGF) activity in the eye. According to REGENXBIO, the three-year data from the ALTITUDE study demonstrated sustained improvements in measures of diabetic retinopathy and reductions in the need for supplemental anti-VEGF treatments among treated participants. The company is continuing clinical development of surabgene lomparvovec across retinal diseases, with ongoing studies evaluating its potential to provide durable treatment following a single administration.
Three-Year ALTITUDE Data Show Treatment Durability
The ALTITUDE Phase II study is evaluating surabgene lomparvovec in patients with diabetic retinopathy. REGENXBIO reported that the latest analysis provides three years of follow-up from participants treated in the study. The company highlighted sustained clinical effects and continued disease control following administration of the investigational gene therapy. Diabetic retinopathy is a complication of diabetes that can damage retinal blood vessels and, in advanced stages, threaten vision. Anti-VEGF therapies are commonly used to manage retinal vascular disease, but treatment generally requires repeated injections over time. A gene therapy capable of producing a sustained biological effect after a single administration could potentially reduce the frequency of treatment required by patients if its safety and efficacy are established in clinical development. The three-year follow-up therefore provides information about the durability of surabgene lomparvovec’s effects.
Surabgene Lomparvovec Targets VEGF Activity
Surabgene lomparvovec is designed to deliver the RGX-314 transgene to retinal cells. The transgene is intended to produce an antibody fragment that inhibits VEGF, a protein involved in abnormal blood-vessel growth and vascular leakage associated with several retinal diseases. By introducing genetic instructions designed to suppress VEGF activity, the therapy aims to provide a sustained biological effect without requiring patients to receive repeated anti-VEGF injections at the same frequency. REGENXBIO is developing surabgene lomparvovec using its NAV® AAV8 vector platform, which is intended to deliver the therapeutic genetic material to relevant cells in the eye. The company is investigating different routes of administration and dosing approaches across its clinical programs to evaluate how the therapy can be delivered safely and effectively.
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Gene Therapy Could Reduce Treatment Burden
One of the potential advantages being investigated with surabgene lomparvovec is the possibility of reducing the treatment burden associated with repeated retinal injections. Diabetic retinopathy can require long-term monitoring and treatment, particularly when vascular leakage or abnormal blood-vessel growth threatens vision. Anti-VEGF therapies can be effective but may require repeated administration depending on disease activity and individual patient response. A one-time gene therapy approach could potentially provide sustained production of an anti-VEGF therapeutic molecule within the eye. The three-year data presented by REGENXBIO are therefore relevant to the company’s objective of determining whether surabgene lomparvovec can maintain its biological and clinical effects over an extended period. However, the therapy remains investigational, and longer-term clinical evaluation will be necessary to establish its overall safety and efficacy. REGENXBIO’s presentation adds to the growing clinical evidence surrounding gene therapy for retinal diseases. The company is studying surabgene lomparvovec in multiple retinal conditions where VEGF plays an important role in disease progression. The latest ALTITUDE findings provide additional follow-up on diabetic retinopathy and allow researchers to assess the persistence of treatment effects several years after administration. Continued monitoring will be important for understanding the long-term safety profile, durability of response and potential need for additional treatment. The findings also contribute to broader research into whether AAV-based gene therapies can provide durable biological effects in chronic retinal diseases.
The three-year ALTITUDE data represent another milestone in REGENXBIO’s clinical development program for surabgene lomparvovec. The reported durability of treatment effects supports continued investigation of the therapy as a potential one-time treatment approach for diabetic retinopathy, while ongoing clinical studies will be needed to establish its benefit-risk profile. As retinal diseases associated with diabetes continue to require long-term management, gene therapy approaches designed to provide sustained anti-VEGF activity are being evaluated as a way to potentially reduce treatment frequency. REGENXBIO’s ongoing studies will provide additional evidence regarding the clinical performance and long-term potential of surabgene lomparvovec.
Source: REGENXBIO press release
