REGENXBIO to present 2.5-year ALTITUDE gene therapy data as NAAVIGATE enrolls
The July ASRS presentation extends two-year dose-level-3 data already tied to no intraocular inflammation, as AbbVie's larger Phase IIb/III NAAVIGATE trial tests the same suprachoroidal dose in a sham-controlled design.
Executive Summary
- REGENXBIO will show longer-term follow-up data on its suprachoroidal gene therapy in diabetic retinopathy, extending a durability and tolerability picture it has already disclosed in earlier form.
- The dose being followed here is the same one AbbVie has now moved into a larger, sham-controlled trial, so this update speaks directly to whether that bigger bet holds up.
- The feeder study has run for more than five years, closed enrollment above its original target, and is not designed to support approval on its own.
- Gene therapy remains a small slice of the diabetic retinopathy treatment landscape, with existing antibody and protein therapies dominating the same VEGF target.
The update
REGENXBIO Inc. said it will present two-and-a-half-year long-term follow-up data from the Phase 2 ALTITUDE trial (NCT04567550) of surabgene lomparvovec, also known as sura-vec or ABBV-RGX-314, at the ASRS 44th Annual Meeting in July 2026. The therapy is a one-time gene therapy that uses an AAV8 vector delivered to the suprachoroidal space of the eye, designed to produce sustained inhibition of vascular endothelial growth factor (VEGF), a protein tied to the leaky new blood vessels that drive diabetic retinopathy. Two-year dose-level-3 data from the same trial, disclosed in the same June 29, 2026 release, showed a durable efficacy profile with no intraocular inflammation observed in 15 patients. REGENXBIOREGENXBIO Announces First Patient Dosed in Phase IIb/III NAAVIGATE Trial of Surabgene Lomparvovec in Diabetic Retinopathy; Company to Receive $100 Million MilestoneJun 29, 2026
Trial identity
ALTITUDE (NCT04567550) is a Phase 2, non-registrational, interventional trial sponsored by AbbVie with REGENXBIO as collaborator, enrolling 139 patients across United States sites. The trial started November 20, 2020 and moved to Active, not recruiting status in July 2025 once it closed enrollment at 139, above its original 40-patient target. Its primary completion date is listed as June 1, 2026, with full study completion set for December 1, 2026. The primary outcome measure is the proportion of participants achieving a 2-step or greater improvement in diabetic retinopathy severity on the ETDRS-DRSS scale at Week 48. NCT04567550RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) With and Without Center Involved-Diabetic Macular Edema (CI-DME)NCT04567550
Enrollment and timeline history
The trial's target grew across several amendments, from 40 to 60 patients in November 2021, to 100 in November 2022, to 130 in March 2025, and finally to 139 in July 2025. Its primary completion date shifted in step, moving from an original May 2021 estimate out to June 2026 across five separate revisions. That pattern reflects a trial that expanded its enrolled population and extended its follow-up window as it added the dose-finding parts described in the ALTITUDE design, tracking with a Phase 2 program built for longer-term durability data rather than a fixed registrational timeline. NCT04567550RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) With and Without Center Involved-Diabetic Macular Edema (CI-DME)NCT04567550
The bigger trial it feeds
REGENXBIO said the first patient has been dosed in the Phase IIb/III NAAVIGATE trial (NCT07592273) of surabgene lomparvovec in diabetic retinopathy, triggering a $100 million milestone payment from AbbVie. NAAVIGATE is a multicenter, randomized, masked, sham-controlled study using the same 1.0x10^12 genome copies per eye dose evaluated as dose level 3 in ALTITUDE, plus short-course topical steroids, with a primary endpoint of greater than 2-step improvement on the DRSS at one year. The Phase IIb portion is expected to enroll approximately 135 participants in the United States. NAAVIGATE's registry listing shows an enrollment target of 576 and a comparator arm using sham and artificial tears, with a primary completion date of June 2028. REGENXBIOREGENXBIO Announces First Patient Dosed in Phase IIb/III NAAVIGATE Trial of Surabgene Lomparvovec in Diabetic Retinopathy; Company to Receive $100 Million MilestoneJun 29, 2026
The competitive field
Gene therapy has been used in only two trials for diabetic retinopathy, making the modality sparse in this specific indication even though VEGF itself is a heavily pursued target across ophthalmology and oncology. Direct comparators sharing both the VEGF-A target and disease area include Regeneron's aflibercept, Roche's faricimab and ranibizumab, and AbbVie's own surabgene lomparvovec program in wet age-related macular degeneration (NCT07007065), alongside Adverum's competing suprachoroidal and subretinal gene therapy candidates for the same eye disease category. Within diabetic retinopathy specifically, the field includes Ocular Therapeutix's OTX-TKI, now in Phase 3 with a DRSS-based endpoint, as the nearest late-stage rival testing a comparable functional outcome.
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