ROCKVILLE, Md., June 29, 2026
REGENXBIO Inc. has reached a major milestone in the advancement of gene therapy for diabetic retinopathy with the first patient dosed in the Phase IIb/III NAAVIGATE clinical trial evaluating surabgene lomparvovec (sura-vec, ABBV-RGX-314) using suprachoroidal delivery. The achievement also triggers a $100 million milestone payment from AbbVie, underscoring the continued progress of the companies’ strategic collaboration to develop innovative one-time gene therapies for chronic retinal diseases. The NAAVIGATE study is designed to evaluate the safety and efficacy of sura-vec in patients with non-proliferative diabetic retinopathy (NPDR) without center-involved diabetic macular edema, addressing one of the leading causes of vision loss among working-age adults worldwide. By combining AAV gene therapy technology with targeted inhibition of the vascular endothelial growth factor (VEGF) pathway, REGENXBIO aims to deliver a durable treatment capable of slowing disease progression through a single in-office administration, potentially reducing the need for repeated anti-VEGF injections and improving long-term visual outcomes for millions of patients.
Phase IIb/III Trial Targets Durable One-Time Treatment
The NAAVIGATE Phase IIb/III trial is a multicenter, randomized, masked, sham-controlled study evaluating surabgene lomparvovec administered through suprachoroidal delivery, a minimally invasive approach designed to efficiently deliver gene therapy to retinal tissues. Approximately 135 participants are expected to enroll in the U.S. Phase IIb portion of the study. The primary endpoint will measure the proportion of patients achieving a greater than two-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) after one year of treatment. Investigators will also assess safety, durability, and long-term disease modification following a single administration.
Unlike conventional therapies that often require lifelong, repeated intravitreal injections, sura-vec is designed as a one-time gene therapy capable of producing sustained anti-VEGF protein expression within the eye. If successful, this innovative treatment strategy could significantly reduce treatment burden while preventing disease progression before irreversible vision loss occurs, representing an important advancement in retinal disease management.
Clinical Data Strengthens Confidence in Gene Therapy Platform
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REGENXBIO also announced plans to present two-and-a-half-year follow-up data from the Phase II ALTITUDE trial during the American Society of Retina Specialists (ASRS) Annual Meeting 2026. Previously reported two-year results demonstrated durable efficacy, favorable tolerability, and no intraocular inflammation among patients treated with the selected dose level, supporting continued development of the therapy. In addition, the company will present five-year long-term follow-up data from its Phase I/IIa study evaluating sura-vec in wet age-related macular degeneration (AMD), further highlighting the long-term potential of its AAV-based retinal gene therapy platform. REGENXBIO also expects to report topline results from the pivotal ATMOSPHERE and ASCENT clinical trials during the fourth quarter of 2026, marking additional milestones in its late-stage ophthalmology pipeline.
The company’s proprietary NAV AAV8 vector technology continues to position REGENXBIO among the leaders in gene therapy innovation, with programs spanning retinal diseases, Duchenne muscular dystrophy, and rare genetic disorders. The initiation of the Phase IIb/III NAAVIGATE trial represents another significant step toward transforming diabetic retinopathy treatment through durable gene therapy, offering the possibility of long-lasting disease control, fewer clinical interventions, and improved quality of life for patients living with progressive retinal diseases while reinforcing the growing role of advanced genetic medicines in modern ophthalmology.
Source: REGENXBIO press release
