ZM-08

Zhongmou Therapeutics has released first-in-human results for an optogenetic gene therapy designed for patients with advanced retinitis pigmentosa, a condition where conventional gene-specific approaches are often ineffective.Early data from the Phase I MOON study, presented at the Advanced Therapies Congress (ATC), showed that 83% of patients treated with ZM-02 achieved at least a 0.3 LogMAR improvement at 36 weeks, with an average gain of 0.59 LogMAR, equivalent to roughly 30 ETDRS letters. Zhongmou noted that all participants have since completed 52-week follow-up, with results demonstrating "sustained" gains in acuity, mobility and color perception for at least one year after a single intravitreal injection.Patients also achieved significant improvements in multi-luminance mobility testing and regained some color perception, the company said. There were no drug-related serious adverse events or dose-limiting toxicities."Advanced retinitis pigmentosa is a disease where gene-specific therapies typically fail due to near-complete photoreceptor loss," stated CEO Yin Shen. By introducing a novel photosensitive protein, called PsCatCh2.0, into retinal ganglion and bipolar cells, ZM-02 doesn't rely on rescuing degenerated photoreceptors, but instead reroutes visual signaling through intact cells. Shen believes this explains why patients were able to switch from complete blindness to regaining functional vision, including "navigation capability in daily environments, and even safely returning to activities such as cycling without using external devices."'Foundational step'She called the results a "foundational step toward redefining what is clinically possible" for patients with little remaining photoreceptor function. Optogenetics has been used in neuroscience research for over 20 years, but Zhongmou says it is among the first to apply the technology clinically for restoring vision.Zhenghong Gao, the company's chief strategy officer, said the MOON study data "underscore how optogenetics is shifting the treatment landscape for patients with advanced retinal degeneration and generating meaningful clinical enthusiasm." ZM-02 secured an orphan drug designation from the FDA in October of last year and an IND submission for a Phase I/II study remains "on track," the company said.While ZM-02 is Zhongmou's most advanced programme, it is also developing ZM-01 for X-linked retinoschisis and ZM-08 for dry age-related macular degeneration, both in preclinical testing.