Zhongmu Healthcare to Initiate a Gene Therapy Clinical Trial for Advanced Retinitis Pigmentosa

Zhongmu Healthcare Technology (Wuhan) Co., Ltd. (“Zhongmu Healthcare”) has obtained approval from the Ethics Committee of Beijing Tongren Hospital affiliated with Capital Medical University (Ethics Approval No.: TREC2024-KY005), and has also completed formal filing with the National Health Commission.

Zhongmu Healthcare plans to conduct an investigator-initiated clinical study at Beijing Tongren Hospital to evaluate ZM-02 for advanced retinitis pigmentosa. The study will assess the safety and tolerability, as well as preliminary efficacy, following a single intravitreal administration of ZM-02. Prof. Wei Wenbin and Prof. Lu Laichun will serve as the principal investigators. As a strategic partner, Paiten Bio is honored to provide GMP-grade plasmids and AAV manufacturing for this program.

Founder’s Statement

Prof. Shen Yin, Founder of Zhongmu Healthcare, said:
“We are pleased that the ZM-02 clinical trial can be conducted at Beijing Tongren Hospital affiliated with Capital Medical University. Zhongmu’s goal is to help RP patients restore visual perception and improve quality of life. We would like to express our sincere gratitude to friends from all sectors for joining hands with Zhongmu to work toward this vision, and we also extend our thanks to Paiten Bio for their strong coordination and substantial support for the ZM-02 program. We have been highly impressed by Paiten Bio’s professionalism—whether in program communication or throughout the manufacturing process, everything has been exceptionally smooth. We look forward to continued collaboration and sustained innovation in the future to bring light and hope to patients with vision function impairment.”

Paiten Bio’s Commitment

Paiten Bio sincerely congratulates the ZM-02 program on smooth progress and looks forward to bringing a glimpse of hope for vision recovery to patients. In the future, Paiten Bio will continue to deepen its development of vector manufacturing processes and technologies, providing one-stop CMC solutions to support the filing of gene therapy products and to accelerate the translation of research outcomes into real clinical impact.

About the ZM-02 Treatment Approach

Retinitis pigmentosa (RP) is a complex inherited ocular disease. Due to a wide variety of genetic mutations, retinal photoreceptor cells undergo degeneration and death, gradually leading to a decline in retinal photoreceptor function and ultimately to progressive vision loss.

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

Through a single intravitreal injection of ZM-02, the gene fragment encoding a photosensitive protein is delivered to retinal cells downstream of photoreceptors that remain viable, enabling retinal cells that are unable to sense light to regain light-sensing capability. The therapy also leverages the preserved visual neural circuitry within the retina to help restore visual function. Importantly, ZM-02 is not limited by specific genetic mutation types associated with retinitis pigmentosa, and is designed as a universal gene therapy approach.

About Zhongmu Healthcare

Established in 2019, Zhongmu Healthcare is an innovative biopharmaceutical company with leading technologies for retinal gene therapy. It has developed an integrated platform covering systematic capabilities for retinal gene therapy R&D—from design and validation to optimization—along with targeting optimization technologies for AAV viral vectors, and a globally leading optogenetics iteration technology platform.

Based on the outcomes and accumulation from more than two decades of research and exploration by Prof. Shen Yin’s team in retinal gene therapy, Zhongmu Healthcare has successfully translated laboratory research findings into clinical application. The company has progressed from single-gene repair to restoring retinal function, and has built a product pipeline centered on patients’ clinical needs. ZM-02, launched by Zhongmu Healthcare, has been produced and prepared by Paiten Bio under GMP conditions. Its initial indication covers advanced retinitis pigmentosa. It is expected to be expanded to other retinal orphan diseases that severely affect visual function, as well as common diseases such as late-stage geographic atrophy (GA) in AMD.