AAV Gene Therapy for Wet AMD Proves Safe and Effective in Bilateral Study

A new study presented at the European Society of Retina Specialists (EURETINA) 2025 conference shows that an AAV gene therapy for wet age-related macular degeneration (wet AMD) is safe and effective when administered to both eyes. This marks the first time a gene therapy for this condition has been evaluated for bilateral treatment.

The study, led by Dr. Dante Pieramici, a retina specialist at California Retina Consultants, included ten patients who received the AAV gene therapy surabgene lomparvovec (also known as sura-vec or ABBV-RGX-314) in their second eye at least one year after the first.

Addressing Immune System Concerns

A key concern with using viral vectors for gene therapies is the potential for an immune response after the initial treatment, which could make subsequent treatments less effective. “We didn’t see any of those problems,” Dr. Pieramici stated. He suggested that the subretinal delivery method, which places the therapy directly under the retina, may mitigate these risks compared to a more common intravitreal injection.

Sura-vec is an AAV8 vector engineered to deliver a gene that produces an antibody fragment. This fragment is designed to inhibit VEGF, a protein that promotes the growth of abnormal, leaky blood vessels associated with wet AMD.

Impressive Clinical Outcomes

The results demonstrated that the therapy successfully maintained stable vision and retinal health in the second eye. On average, patients experienced a 92% reduction in the need for anti-VEGF injections, going from 8.8 annual injections down to just 0.7. At the one-year mark, 60% of the second eyes were completely injection-free.

No serious adverse events were reported. Minor side effects, such as a temporary conjunctival hemorrhage and retinal pigmentation changes, were observed but resolved quickly.

Dr. Dina Zur, president of the Israeli Retina Society, commented that the data is “very encouraging” and represents an important step toward investigating bilateral treatment for wet AMD patients. She noted that the safety profile, with no new safety signals observed, is particularly significant.

About ABBV-RGX-314

ABBV-RGX-314 is an AAV8 gene therapy co-developed by REGENXBIO and AbbVie as a potential one-time treatment for chronic retinal conditions like wet AMD. It works by delivering a gene that produces an antibody fragment to inhibit the VEGF pathway, which causes the growth of new, leaky blood vessels. REGENXBIO is advancing research on two delivery methods: a subretinal procedure and a suprachoroidal injection.