Regenxbio announced the publication of results from its phase 1/2a trial evaluating the safety and tolerability of a single dose of subretinal ABBV-RGX-314 for the treatment of wet AMD. Two-year data were published in The Lancet, in a paper titled “Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study.”

These study results demonstrated that a single administration of ABBV-RGX-314 was generally well tolerated. Stable or improved VA and retinal thickness were observed with few or no supplemental anti-VEGF injections in most patients at 2 years. Patients who received therapeutic doses demonstrated sustained levels of ABBV-RGX-314 protein and stable or improved vision and retinal anatomy with few-to-no supplemental anti-VEGF injections in most participants up to 2 years. Regenxbio has also reported additional positive interim data from a long-term follow-up study of ABBV-RGX-314, which indicate that treatment continues to be well tolerated and demonstrates long-term, durable treatment effect up to 4 years.