RGX-314 gene therapy shows promise in treating wet AMD in Phase 1/2a study

RGX-314, a novel gene therapy, for the treatment of neovascular age-related macular degeneration (nAMD) shows promising results in terms of safety and efficacy, according to results of a Phase 1/2a dose-escalation study published in The Lancet.

Researchers found that subretinal delivery of RGX-314 was generally well-tolerated with no clinically recognized immune responses. Higher doses of RGX-314 resulted in sustained suppression of vascular endothelial growth factor A (VEGF-A), stable or improved vision, and reduced the need for supplemental anti-VEGF injections.

This suggests that RGX-314 has the potential to offer sustained VEGF-A suppression, control exudation, maintain vision, and reduce treatment burden after a single administration, thus representing a significant advancement in the management of nAMD.

“We have started 2024 with strong, positive new data from the ABBV-RGX-314 program, and we believe that there is multi-billion-dollar potential for ABBV-RGX-314 to become a first-in-class gene therapy for wet AMD and the standard of care to treat and prevent progression of diabetic retinopathy,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO in a press release. “To have these Phase I/IIa data published in The Lancet highlights the groundbreaking work of our scientists and investigators, and further validates the clinically transformative nature of ABBV-RGX-314 as a potential one-time gene therapy for wet AMD that may help patients maintain or improve their vision.”

Reference
Campochiaron PA, Avery R, Brown D, et al. Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study. Lancet. 2024;DOI:https://doi.org/10.1016/S0140-6736(24)00310-6