Gene Therapy for Wet AMD: Phase 1/2 Clinical Trial Results

Wednesday, December 11 2024 | 10 h 01 min | News

A new study published in The Lancet shared results from a Phase 1/2 clinical trial testing a one-time anti-VEGF gene therapy for wet age-related macular degeneration (wet AMD). The Johns Hopkins University study followed 42 patients for 2 years after gene therapy treatment.

Injected RGX-314 gene therapy produces an anti-VEGF drug called ranibizumab (Lucentis), which is a commonly used therapy for wet AMD. Retinal injection allows cells to make their own anti-VEGF medicine and hopefully reduces or eliminates the need for regular injections. In this study, there was a 60-80% reduction in the number of injections that patients needed. After injection of the gene therapy, patients received 1-4 injections per year compared to about 10 injections before gene therapy.

The treatment is now being tested in two larger Phase 3 clinical trials to ensure that it is safe and effective.