Zhongmou Therapeutics to Initiate Gene Therapy Clinical Trial for Advanced Retinitis Pigmentosa

Zhongmou Therapeutics has received approval from the Ethics Committee of Beijing Tongren Hospital, Capital Medical University, under approval number TREC2024-KY005, and has completed official filing with the National Health Commission of China.

Zhongmou Therapeutics plans to conduct an investigator-initiated clinical study of gene therapy for advanced retinitis pigmentosa at Beijing Tongren Hospital, Capital Medical University. The study will evaluate the safety, tolerability, and preliminary efficacy of ZM-02 in patients with retinitis pigmentosa through a single intravitreal administration. Professor Wei Wenbin and Professor Lu Laichun of Beijing Tongren Hospital will serve as the principal investigators. PackGene Biotech is honored to provide GMP-grade plasmid and AAV manufacturing services for this project.

Professor Shen Yin, founder of Zhongmou Therapeutics, said: “We are very pleased that the clinical study of ZM-02 will be conducted at Beijing Tongren Hospital, Capital Medical University. Zhongmou’s goal is to help patients with RP restore visual perception and improve their quality of life. We are grateful to all partners and friends who have joined hands with Zhongmou to work toward this vision, and we also sincerely thank PackGene Biotech for its close collaboration and strong support for the ZM-02 project. PackGene Biotech’s professionalism is evident to all of us. Both project communication and the entire manufacturing process have proceeded very smoothly. We look forward to continuing our collaboration and innovation in the future, bringing light and hope to patients with visual dysfunction.”

PackGene Biotech sincerely wishes the ZM-02 project smooth progress and hopes it will bring the dawn of vision restoration to patients. Looking ahead, PackGene Biotech will continue to deepen its development of vector processes and technologies, provide one-stop CMC solutions for drug innovation, advance gene therapy drug applications, and support the translation of scientific research achievements.

About the ZM-02 Therapeutic Approach

Retinitis pigmentosa is a complex inherited ophthalmic disease caused by a wide range of genetic mutations that lead to the degeneration and death of retinal photoreceptor cells. This results in a gradual decline in the retina’s light-sensing function and ultimately causes vision loss.

Optogenetic gene therapy is a cutting-edge technology that can restore the retina’s light-sensing function by expressing light-sensitive proteins in downstream retinal cells. ZM-02 is a novel retinal gene therapy developed based on optogenetic technology.

Through a single intraocular injection of ZM-02, this therapy delivers gene fragments encoding light-sensitive proteins into surviving retinal cells downstream of photoreceptors, enabling retinal cells that originally lacked light sensitivity to acquire light-sensing function. At the same time, it leverages the remaining visual circuits in the retina to restore visual function. This therapy is not limited by specific types of genetic mutations associated with retinitis pigmentosa, making it a broadly applicable gene therapy technology.

About Zhongmou Therapeutics

Founded in 2019, Zhongmou Therapeutics is an innovative drug company with leading technologies in retinal gene therapy. The company has established a comprehensive platform covering systematic technologies for the design, validation, optimization, and development of gene therapies for various retinal diseases; AAV viral vector targeting optimization technologies; and world-leading iterative optogenetics technologies.

Based on more than 20 years of exploration and accumulated achievements by Professor Shen Yin’s team in gene therapy technologies for ophthalmic diseases, Zhongmou Therapeutics has successfully translated laboratory research into clinical application. The company has advanced from single-gene repair to retinal function restoration and established a product pipeline centered on clinical patient needs. ZM-02, which is being launched in this study, was manufactured under GMP conditions by PackGene Biotech. Its indication covers advanced retinitis pigmentosa and will later be expanded to other ophthalmic rare diseases that severely affect visual function, as well as common diseases such as advanced dry geographic atrophy AMD.