Zhongmou’s ZM-02 Gene Therapy Shows Durable Vision Gains in Retinitis Pigmentosa

Zhongmou Therapeutics’ gene therapy, ZM-02, demonstrated promising efficacy in a first-in-human trial for advanced retinitis pigmentosa (RP). In the single-site MOON study (NCT06292650) in Beijing, 83% of patients achieved ≥0.3 LogMAR improvement in best-corrected visual acuity (BCVA) at 36 weeks, with a mean gain of 0.59 LogMAR (~30 ETDRS letters). Improvements were also seen in low-light mobility and colour vision. Importantly, these gains were maintained at the 52-week follow-up after a single dose, with no drug-related serious adverse events reported.

ZM-02, an AAV vector-based therapy, delivers a light-sensitive protein gene via ocular injection. RP, a rare retinal degenerative disease, has a sparse treatment landscape, with Luxturna being the only approved gene therapy globally. Several pipeline candidates, including Ocugen’s OCU400 and Beacon Therapeutics’ laru-zova, are in advanced development, highlighting growing momentum in RP therapeutics.

05-12-2025