Gene Therapy for Wet AMD – A Potential New Approach

People living with wet age-related macular degeneration (AMD) continue to need new and better treatment options. The current standard of care requires frequent injections of anti-VEGF (anti-vascular endothelial growth factor) via a needle into the eye. These injections are uncomfortable and are required every 4-16 weeks, which makes sticking to the treatment hard for many living with wet AMD. Without these chronic treatments, people impacted by wet AMD will continue to experience vision loss which cannot be recovered.

The National Eye Institute (NEI) recently announced a redefined mission¹ that includes a call for continued innovation in new solutions for communities long in need of more impactful treatments. Gene therapy is currently being researched as one of these potential new treatment options for wet AMD.

While wet AMD is not a genetic disorder, gene therapy may help people with wet AMD by delivering a gene which can instruct the cells in the eye to continuously produce an anti-VEGF protein. Gene therapy for treating wet AMD is designed to be given as a one-time administration with the goal of decreasing or eliminating the need for frequent anti-VEGF injections.

Several clinical trials are underway to investigate gene therapy as an approach for treating wet AMD. REGENXBIO, a leading clinical-stage biotechnology company, is currently enrolling participants in two active gene therapy trials for wet AMD. Individuals and caregivers should talk to their doctor about whether a clinical trial might be right for them or their loved one. You can also visit Clinical Trials | BrightFocus Foundation or email patientadvocacy@regenxbio.com to learn more about specific trials.

¹https://www.nei.nih.gov/sites/default/files/2021-10/NEI-StrategicPlan-ExecutiveSummary-508c.pdf