PUMCH-E111

PUMCH-E111 is an investigational gene therapy developed by Peking Union Medical College Hospital for the treatment of inherited retinal dystrophy (IRD) caused by mutations in the *RLBP1* gene. The therapy is designed to deliver a functional copy of the *RLBP1* gene to the retina via a single intravitreal injection. *RLBP1* encodes the cellular retinaldehyde-binding protein (CRALBP), which is essential for the regeneration of visual pigments in the retinal pigment epithelium and Mueller cells. By restoring CRALBP function, PUMCH-E111 aims to improve or stabilize vision in patients with conditions such as Bothnia dystrophy or retinitis punctata albescens. It is currently being evaluated in a Phase 1/2 dose-escalation study to assess safety, immunogenicity, and preliminary efficacy.