One-time gene therapy surabgene lomparvovec (sura-vec) demonstrated durable vision benefits and favorable long-term safety in wet AMD and diabetic retinopathy, with Phase III wet AMD topline data expected in Q4 2026.
Written By: Mayuresh Salvi, PharmD
Reviewed By: Pharmacally Editorial Team
A one-time investigational gene therapy, surabgene lomparvovec (sura-vec, ABBV-RGX-314), demonstrated durable long-term efficacy and a favorable safety profile in patients with wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR), according to data presented at the American Society of Retina Specialists (ASRS) 2026 Annual Meeting. The findings suggest the therapy could substantially reduce the need for frequent anti-vascular endothelial growth factor (anti-VEGF) injections while maintaining long-term visual outcomes. These findings were presented at a scientific meeting and have not yet been published in a peer-reviewed journal.
Disease Background
Wet AMD is a leading cause of irreversible vision loss in older adults and is characterized by abnormal, leaky blood vessels beneath the retina. Although anti-VEGF therapies have transformed treatment, most patients require lifelong intravitreal injections every 4 to 8 weeks, and poor adherence often leads to vision loss. Diabetic retinopathy, another major cause of blindness among working-age adults, can progress from non-proliferative diabetic retinopathy (NPDR) to vision-threatening complications, highlighting the need for durable, disease-modifying therapies.
Mechanism of Action
Surabgene lomparvovec is an adeno-associated virus serotype 8 (AAV8)-based gene therapy designed to deliver the genetic code for an anti-VEGF antibody fragment directly to retinal cells. The therapy is intended to provide sustained intraocular VEGF inhibition after a single administration, potentially reducing or eliminating the need for repeated anti-VEGF injections.
Clinical Trial Overview
The five-year wet AMD data were derived from Cohorts 3 and 4 of an ongoing Phase I/IIa study evaluating subretinal administration of sura-vec at doses similar to those used in the pivotal Phase III ATMOSPHERE (NCT04704921) and ASCENT (NCT05407636) trials. Together, these studies have enrolled more than 1,200 participants across over 200 sites and compare sura-vec with ranibizumab and aflibercept, respectively.
Investigators also reported 2.5-year follow-up results from the Phase II ALTITUDE trial (NCT04567550), which evaluated suprachoroidal administration of sura-vec in patients with NPDR. The same dose is currently being studied in the ongoing Phase IIb/III NAAVIGATE trial (NCT07592273).
Long-Term Efficacy and Safety
Among patients with wet AMD who had previously required frequent anti-VEGF injections, a single subretinal administration of sura-vec maintained stable or improved best-corrected visual acuity (BCVA) through five years while substantially reducing the need for supplemental anti-VEGF treatment. Compared with matched real-world patients receiving conventional anti-VEGF therapy, sura-vec recipients demonstrated better preservation of vision, reduced fluctuations in central retinal thickness, and significantly fewer injections. Long-term follow-up identified no new safety signals, and no intraocular inflammation was observed despite the absence of prophylactic steroid treatment.
In the ALTITUDE study, a single suprachoroidal administration of sura-vec produced durable clinical benefit through 2.5 years. More than half of participants (55%) achieved a greater than two-step improvement on the Diabetic Retinopathy Severity Scale without additional treatment, while 70% experienced no vision-threatening events. Most patients who achieved a one-step improvement at one year subsequently improved to a two-step response by 2.5 years, supporting the therapy’s potential to modify the underlying disease. Sura-vec remained well tolerated, with no intraocular inflammation observed through 2.5 years in participants receiving short-course prophylactic topical corticosteroids.
Clinical Implications
Commenting on the findings, Steve Pakola, MD, Chief Medical Officer of REGENXBIO, said the durable efficacy observed across multiple routes of administration supports the potential of sura-vec to preserve vision while reducing the burden of chronic anti-VEGF injections. He noted that the five-year wet AMD results were generated in patients who previously required frequent anti-VEGF therapy, representing a more challenging population than treatment-naïve patients evaluated in some other studies.
Path Forward
REGENXBIO and AbbVie expect to report topline results from the pivotal Phase III ATMOSPHERE and ASCENT trials in the fourth quarter of 2026. Positive findings could support future regulatory submissions for wet AMD, while results from the ongoing NAAVIGATE study will further define sura-vec’s development pathway in diabetic retinopathy.
Reference
About the Writer
Mayuresh Sunil Salvi (Linkedin) is a PharmD professional and healthcare writer with a strong interest in pharmacovigilance, drug safety, and emerging medical research. He is passionate about exploring new drug discoveries, clinical research, and advances in evidence-based medicine. His interests also include ward rounds, prescription audits, and treatment analysis to support rational pharmacotherapy and improved patient care.