ZM-02

ZM-02 is an optogenetic gene therapy developed by Zhongmou Therapeutics for the treatment of advanced retinitis pigmentosa (RP). The therapy utilizes an adeno-associated virus (AAV) vector to deliver a gene encoding an engineered photosensitive protein called PsCatCh2.0 (or PsCaCh2.e), which is derived from channelrhodopsin found in marine algae. Administered via a single intravitreal injection, ZM-02 is designed as a mutation-agnostic treatment that bypasses degenerated photoreceptors and reactivates residual inner retinal circuits, enabling surviving retinal cells to respond to light. In the first-in-human MOON trial conducted in China, the therapy demonstrated significant clinical improvements in visual acuity at 36 weeks, with some patients regaining color vision. ZM-02 has received FDA Orphan Drug Designation and IND clearance to proceed with Phase 1/2 clinical trials in the United States.

Other names

PsCatCh2.0PsCatCh-2.0PsCatCh 2.0PsCaCh2.ePsCaCh-2.ePsCaCh 2.e