Retinitis pigmentosa caused by biallelic mutations in the RLBP1 gene research hub

An open-label first-in-human single ascending dose study to explore the safety, tolerability and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa caused by mutations in the RLBP1 gene

This study is testing a new gene therapy, CPK850, for a serious eye condition called retinitis pigmentosa caused by specific gene changes. It's the first time this treatment is being given to people, checking if it's safe, how well it's tolerated, and if it helps improve vision.