Evaluating the efficacy of a gene therapy to treat vision loss
The purpose of this study is to evaluate the efficacy and safety of a novel gene therapy in participants with wet age-related macular degeneration (nAMD), a significant cause of vision loss for people worldwide. Current standard of care therapies require life-long intraocular injections that are typically repeated every 4-12 weeks to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. As such, gene therapy RGX-314 is being developed as a potential one-time treatment for nAMD.