Zhongmou Therapeutics Reports First-in-Human Success of Optogenetic Gene Therapy ZM-02 for Retinitis Pigmentosa
Zhongmou Therapeutics has developed ZM-02, a next-generation optogenetic gene therapy based on an adeno-associated virus (AAV) vector delivering the novel photosensitive protein gene PsCatCh2.0.
ZM-02 is designed to treat advanced retinal degenerations including retinitis pigmentosa (RP), irrespective of causative gene mutations.
The company presented first-in-human clinical results demonstrating that ZM-02 can help RP patients partially restore vision, effectively enabling them to see again.
ZM-02 requires significantly lower light intensity than previous therapies, enhancing its safety and effectiveness.
Zhongmou completed early phase 1 clinical trials with a dose-escalation protocol to establish safety and efficacy in RP patients.
The therapy works by targeting retinal ganglion cells (RGCs) with the photosensitive protein, bypassing damaged photoreceptors.
Zhongmou has received orphan drug designation from the U.S. FDA for ZM-02, highlighting its potential impact.
The company recently raised tens of millions of RMB to accelerate global expansion in ophthalmic gene therapy.
ZM-02 is part of a growing pipeline of optogenetic treatments that aim to restore photosensitivity independently of specific genetic mutations, distinguishing itself from gene replacement therapies.
This breakthrough provides hope for patients with advanced vision loss from retinal degenerations who currently have limited treatment options.