ROCKVILLE, Md., July 18, 2026
REGENXBIO Inc. has announced positive long-term clinical data for its investigational one-time gene therapy, surabgene lomparvovec (sura-vec, ABBV-RGX-314), demonstrating durable efficacy and a favorable safety profile in patients with wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR). The results, presented at the American Society of Retina Specialists (ASRS) 44th Annual Meeting in Montreal, highlight the potential of a single gene therapy treatment to provide sustained vision preservation while significantly reducing the need for frequent anti-VEGF injections. The findings support the ongoing late-stage development of sura-vec, with topline results from the pivotal ATMOSPHERE® and ASCENT® Phase III studies expected in the fourth quarter of 2026. The latest data further strengthen the therapy’s position as one of the most promising investigational gene therapies targeting chronic retinal diseases.
Five-Year Data Demonstrate Durable Vision Preservation
The newly reported long-term findings showed that patients with wet AMD maintained stable or improved vision for up to five years following a single administration of sura-vec, despite requiring frequent anti-VEGF injections before receiving treatment. Importantly, investigators reported no drug-related intraocular inflammation (IOI) throughout long-term follow-up in patients treated without prophylactic steroids, reinforcing the therapy’s encouraging safety profile. Additional external control analyses presented at ASRS demonstrated that patients receiving sura-vec preserved vision more effectively than matched real-world patients receiving standard anti-VEGF injections while requiring substantially fewer supplemental treatments. These findings suggest that sura-vec has the potential to address one of the greatest challenges in retinal care by reducing the treatment burden associated with lifelong intravitreal injections while maintaining durable visual outcomes. The investigational therapy is currently being evaluated in the global ATMOSPHERE® and ASCENT® pivotal trials, which have enrolled more than 1,200 participants across over 200 clinical sites worldwide.
Gene Therapy Shows Promising Results in Diabetic Retinopathy
REGENXBIO also reported encouraging 2.5-year follow-up data from the Phase II ALTITUDE® trial evaluating sura-vec in patients with non-proliferative diabetic retinopathy (NPDR). More than 55% of participants achieved greater than a two-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) without requiring additional treatment, while 70% experienced no vision-threatening events during follow-up. The therapy continued to demonstrate a durable safety profile, with no intraocular inflammation observed among treated participants receiving a short course of prophylactic topical steroids. Investigators also noted that many patients who initially achieved a one-step improvement progressed to an even greater two-step improvement over time, suggesting that sura-vec may modify the underlying disease rather than simply controlling symptoms. These data reinforce the potential for a single in-office gene therapy to provide long-lasting protection against disease progression and vision loss in diabetic retinopathy patients.
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Late-Stage Development Advances Toward Pivotal Readouts
Surabgene lomparvovec is an investigational AAV8 gene therapy designed to deliver sustained production of an anti-VEGF antibody fragment following a one-time administration, offering the possibility of long-term disease control without repeated injections. The therapy is being developed for wet AMD, diabetic retinopathy, and other chronic retinal disorders through a collaboration between REGENXBIO and AbbVie. With encouraging long-term efficacy, sustained safety findings, and multiple pivotal studies progressing toward completion, sura-vec represents one of the most advanced gene therapy programs in ophthalmology. The anticipated Phase III topline results later in 2026 could mark a major milestone in the evolution of retinal disease treatment and potentially introduce a transformative new therapeutic option capable of preserving vision while dramatically reducing treatment burden for millions of patients worldwide.
Source: REGENXBIO press release
