ZM-01

ZM-01 is an investigational adeno-associated virus (AAV)-based optogenetic gene therapy developed by Zhongmou Therapeutics for the treatment of X-linked retinoschisis (XLRS). XLRS is a rare inherited retinal disease caused by mutations in the RS1 gene, which leads to the splitting of retinal layers and progressive vision loss. ZM-01 utilizes an AAV vector to deliver a channelrhodopsin gene into retinal cells. Channelrhodopsins are light-gated ion channels that, when expressed, render the remaining retinal cells light-sensitive. This optogenetic approach aims to bypass damaged photoreceptors and restore visual function by turning other retinal cells into functional light sensors. The therapy is administered as a single unilateral intravitreal injection.

Other names

rAAV-hRS1rAAV-hRS-1rAAV-hRS 1