June 29, 2026 —
REGENXBIO announced that the first patient has been dosed in NAAVIGATE, a Phase 2b/3 pivotal clinical trial evaluating surabgene lomparvovec, or sura-vec, also known as ABBV-RGX-314, for nonproliferative diabetic retinopathy without center-involved diabetic macular edema.
The first dosing triggered a $100 million milestone payment from AbbVie under the companies’ existing collaboration. The milestone marks the transition of the sura-vec diabetic retinopathy program into pivotal-stage development following encouraging two-year data from the earlier Phase 2 ALTITUDE trial.
Diabetic retinopathy is a leading cause of vision loss among working-age adults and affects millions of patients worldwide. Current treatment relies largely on repeated anti-VEGF intravitreal injections, which can reduce disease progression but require frequent administration and long-term adherence. In real-world practice, treatment burden, injection anxiety, and under-treatment can limit outcomes.
Sura-vec is designed as a one-time ocular gene therapy that uses the NAV AAV8 vector to deliver a gene encoding an anti-VEGF antibody fragment directly into retinal tissue. The goal is to enable the eye to produce sustained VEGF inhibition after a single administration, potentially reducing the need for repeated injections.
In NAAVIGATE, sura-vec is delivered through the suprachoroidal route, a minimally invasive in-office approach that places the vector between the choroid and sclera without entering the vitreous cavity. This delivery method may offer advantages in biodistribution, procedural practicality, and tolerability compared with traditional intravitreal or subretinal approaches.
The NAAVIGATE study is a multicenter, randomized, masked, sham-controlled trial enrolling patients with nonproliferative diabetic retinopathy without center-involved diabetic macular edema. Participants will receive sura-vec at 1.0 × 10¹² genome copies per eye, the dose evaluated as dose level 3 in the ALTITUDE trial, along with short-course topical prophylactic steroids.
The primary endpoint is a greater than two-step improvement on the Diabetic Retinopathy Severity Scale, or DRSS, at one year. The Phase 2b portion, operationalized by REGENXBIO, is expected to enroll approximately 135 participants in the United States.
The pivotal trial follows two-year ALTITUDE data showing that sura-vec at dose level 3 demonstrated a durable efficacy profile and was well tolerated, with no intraocular inflammation observed in 15 patients. Earlier ALTITUDE results also suggested improvement in diabetic retinopathy severity and reduced treatment burden, supporting the therapeutic concept of sustained anti-VEGF expression after one-time gene therapy administration.
REGENXBIO plans to present two-and-a-half-year ALTITUDE data at the American Society of Retina Specialists 44th Annual Meeting in July 2026.
Sura-vec is also being evaluated for wet age-related macular degeneration through subretinal delivery in the pivotal ATMOSPHERE and ASCENT trials, with topline data expected from both studies in the fourth quarter of 2026. Five-year long-term follow-up data from the Phase 1/2a wet AMD subretinal program are also expected to be presented at ASRS.
If successful, sura-vec could represent a new treatment paradigm for diabetic retinopathy by shifting management from chronic repeat injections toward a one-time, sustained anti-VEGF gene therapy approach delivered in an office-based setting.
