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ZM-01-L
- Development stage
- Phase 1
- Lead developer
- Zhongmou Therapeutics
- Modality
- Gene Therapies
- Administration
- Intravitreal Injection
01
Overview
ZM-01-L is an investigational gene therapy developed for the treatment of X-linked retinoschisis (XLRS), a rare inherited retinal disease caused by mutations in the RS1 gene. The therapy utilizes an adeno-associated virus (AAV)-based vector to deliver a functional copy of the RS1 gene via intravitreal injection, aiming to restore or improve retinal function in affected individuals. ZM-01-L is currently being evaluated in Phase 1 clinical trials, with no approved indications or known adverse events reported as of now. The developer is Zhongmou Therapeutics, and clinical activity has been noted in China[1][2].
- Other names
- zm-01-lzm01-lzm 01-l
02
Targets
RS1 (Retinoschisin)
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