Menu
[California, USA, and CHINA, November 1, 2024] Zhongmou Therapeutics ("Zhongmou"), a global clinical-stage genomic medicine company bringing life-changing treatment to reverse or prevent blindness, today announces that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for ZM-02 optogenetic therapy to treat patients with retinitis pigmentosa (RP) without specific genetic restrictions. ZM-02, a first-in-class optogenetic therapy product candidate, introduces a novel photosensitive protein gene into the retinal cells to convert light, thereby restoring sight.
"We are thrilled that the U.S. FDA has granted ODD to ZM-02, our independently developed optogenetic gene therapy targeting retinitis pigmentosa, an incurable genetic disorder,” said Professor Yin Shen, Founder and CEO of Zhongmou. “This milestone underscores our dedication to advancing innovative gene therapy treatments for ocular diseases. The recognition by the FDA affirms the clinical potential of ZM-02 and propels us forward in our vision of 'making the world not blind' to combat global vision loss. We remain dedicated to pioneering innovations in the field of ocular diseases, always believing in the power of light.”
Professor Yin Shen, Founder and CEO of Zhongmou
The FDA grants Orphan Drug Designation (ODD) to drugs or biologics intended to treat a rare disease or condition affecting fewer than 200,000 people in the United States. The U.S. Orphan Drug Act provides companies that obtain ODD with certain incentives, such as up to 25% clinical trial tax credits for their drug candidates upon approval, prescription drug user fee waivers, and eligibility for accelerated review for approval. If a product receives Orphan Drug Status from the FDA, that product is entitled to 7 years of market exclusivity for the disease in which it has Orphan Drug designation, which is independent of intellectual property protection.
Retinitis Pigmentosa (RP) is the most common inherited retinal disease, caused by mutations in over 270 genes. Affecting more than 2 million people globally with an incidence of about 1 in 3,500, RP leads to progressive degeneration of retinal photoreceptors – initially rods, followed by cones. Onset typically occurs during adolescence, beginning with night blindness, progressing to peripheral vision loss, and eventually resulting in ‘tunnel vision’ and severe vision impairment or blindness. Complications, such as cataracts and macular edema, can worsen visual outcomes. The disease course varies, with some retaining partial vision for years while others progress to blindness before age 40. Currently, there is no effective treatment for retinitis pigmentosa.
ZM-02 is an optogenetic gene therapy developed by Zhongmou Therapeutics to treat retinal degeneration. A single intravitreal injection delivers a novel photosensitive protein gene to retinal cells, enabling them to respond to light despite photoreceptor loss. Unlike traditional gene therapies that target specific mutations, ZM-02 offers a universal approach to addressing multi-gene mutations and other retinal diseases involving photoreceptor degeneration, such as age-related macular degeneration (AMD).
ZM-02 employs newly developed photosensitive proteins with enhanced light sensitivity, safety, and faster response. Preclinical studies have shown stable expression in mouse retinal tissue and restoration of light sensitivity and visual acuity in RP mouse models. A first-in-human trial (NCT06292650) at Beijing Tongren Hospital has demonstrated significant vision improvement and quality of life in RP patients.
Zhongmou Therapeutics is a global clinical-stage biotechnology company focused on restoring vision for millions affected by blindness-causing ocular diseases. Leveraging 20+ years of pioneering gene therapy research, Zhongmou has advanced from lab research to clinical applications, delivering sight-restoring breakthroughs. The company utilizes a robust AAV vector platform and cutting-edge optogenetic therapies to develop a diverse product pipeline targeting significant unmet needs. Zhongmou's innovations include China's first gene therapy for retinal splitting and leading optogenetic therapies featuring photosensitive proteins, solidifying its leadership in the ophthalmic field. These groundbreaking therapies push the boundaries of science and aim to improve public health outcomes through advanced medical innovation.