UGX-202

UGX-202 is an investigational adeno-associated virus (AAV)-based optogenetic gene therapy designed for the treatment of advanced, non-syndromic retinitis pigmentosa (RP). Developed by Shanghai Ugene Biotechnology in collaboration with the Fudan University Affiliated Eye, Ear and Nose Hospital, the therapy utilizes an AAV vector to deliver a gene encoding a light-sensitive protein (opsin) to the retina via intravitreal injection. This approach aims to restore visual function by making surviving retinal cells, such as retinal ganglion cells or bipolar cells, light-sensitive, thereby bypassing the degenerated photoreceptors characteristic of late-stage RP. UGX-202 is currently being evaluated in clinical trials in China to assess its safety, tolerability, and preliminary efficacy in patients with profound vision loss.