REGENXBIO has dosed the first patient in the Phase IIb/III NAAVIGATE trial of surabgene lomparvovec (ABBV‑RGX‑314) for non‑proliferative diabetic retinopathy, triggering a $100M milestone payment from AbbVie and advancing a potential one‑time gene therapy for vision loss.
Written By: Anamika Koshti, PharmD
Reviewed By: Pharmacally Editorial Team
REGENXBIO has advanced investigational gene therapy surabgene lomparvovec (sura-vec, ABBV-RGX-314) into the Phase IIb/III NAAVIGATE trial (NCT07592273) for non-proliferative diabetic retinopathy (NPDR) without center-involved diabetic macular edema (CI-DME), marking the start of late-stage clinical development. The first patient has now been dosed, triggering a $100 million milestone payment from development partner AbbVie and moving the one-time gene therapy a step closer to potential regulatory approval.
One-Time Gene Therapy Targets a Major Cause of Vision Loss
Diabetic retinopathy remains the leading cause of vision loss among adults aged 24 to 75 years worldwide. In the United States alone, nearly 10 million people are affected. As the disease progresses from NPDR to proliferative diabetic retinopathy (PDR), patients face an increasing risk of diabetic macular edema, abnormal retinal blood vessel growth, and irreversible blindness.
Current management often relies on regular monitoring or repeated intravitreal anti-VEGF injections, while advanced disease may require laser photocoagulation or surgery. These repeated interventions can create a significant treatment burden and contribute to undertreatment.
Surabgene lomparvovec uses REGENXBIO’s NAV® AAV8 vector to deliver genetic instructions that enable retinal cells to continuously produce an anti-VEGF antibody fragment after a single administration. Sustained VEGF inhibition could reduce abnormal blood vessel formation and retinal fluid accumulation while minimizing the need for frequent injections.
NAAVIGATE Advances into Late-Stage Development
The Phase IIb/III NAAVIGATE study is evaluating the safety and efficacy of a single suprachoroidal administration of sura-vec at 1.0 × 10¹² genome copies per eye, the same dose previously investigated as dose level 3 in the Phase II ALTITUDE® study (NCT04567550). Participants also receive a short course of topical corticosteroids to reduce post-procedure inflammation.
The trial plans to enroll approximately 135 patients across U.S. sites. Its primary endpoint is the proportion of participants achieving a greater than two-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) after one year.
The program builds on two-year findings from the ALTITUDE study, where dose level 3 demonstrated durable clinical activity and a favorable safety profile. Among 15 evaluable patients, investigators reported no cases of intraocular inflammation, supporting continued development of the in-office gene therapy approach.
Long-Term Retinal Data Expected at ASRS 2026
REGENXBIO will present two-and-a-half-year follow-up data from the ALTITUDE study during the American Society of Retina Specialists (ASRS) 44th Annual Meeting in July 2026. The company will also report five-year follow-up results from its Phase I/IIa study evaluating subretinal sura-vec in wet age-related macular degeneration (AMD) (NCT03066258).
Looking ahead, REGENXBIO and AbbVie expect to announce topline results from the pivotal ATMOSPHERE® (NCT04704921) and ASCENT® ( NCT05407636) trials evaluating subretinal sura-vec for wet AMD during the fourth quarter of 2026.
Chief Medical Officer Steve Pakola, M.D., said advancing NAAVIGATE reflects the companies’ commitment to developing a one-time treatment that may prevent disease progr ession before irreversible vision loss occurs. Arshad Khanani, M.D., M.A., FASRS, Director of Clinical Research at Sierra Eye Associates, added that an in-office, single-administration therapy could improve long-term outcomes while making earlier intervention more practical for patients.
What This Means for Patients
Many people with diabetic retinopathy require years of monitoring or repeated anti-VEGF injections to slow disease progression. If surabgene lomparvovec confirms its efficacy and safety in the ongoing Phase IIb/III trial, it could offer a durable, one-time treatment that reduces the need for frequent procedures while helping preserve vision before irreversible retinal damage develops. Regulatory approval will depend on successful completion of the NAAVIGATE study and review by health authorities.
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About the Writer
Anamika Koshti (LinkedIn) is a PharmD professional and healthcare writer with interests in clinical research, pharmacovigilance, and evidence-based medicine. She has authored peer-reviewed publications on Alzheimer’s disease and PCOS, presented research at national conferences, and gained hands-on experience in medical content development and clinical data interpretation. She is committed to translating complex medical research into accurate, accessible content for healthcare professionals and patients.