Zhongmou Therapeutics’ gene therapy for advanced retinitis pigmentosa (RP) has shown promise in a first-in-human trial.
In the MOON study (NCT06292650) treatment with ZM-02 led to a ≥0.3 logarithm of the minimum angle of resolution (LogMAR) best-corrected visual acuity (BCVA) improvement in 83% of treated patients at 36 weeks, with a mean gain of 0.59 LogMAR. This equates to an improvement of approximately 30 early treatment diabetic retinopathy study (ETDRS) letters.
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Significant improvements were also reported in multi-luminance mobility testing, including low-light navigation. Patients saw a recovery of real-world navigation ability and restoration of colour vision.
While this data is at the 36-week time point, Zhongmou added that the durability of improvement has continued at the 52-week follow-up after a single dose of ZM-02.
The study also showed no drug-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs).
Zhongmou’s chief strategy officer Dr Zhenghong Gao said: “The ZM-02 FIH data underscore how optogenetics is shifting the treatment landscape for patients with advanced retinal degeneration and generating meaningful clinical enthusiasm across the field.”
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By GlobalDataThe study enrolled 12 patients at one site in Beijing, China, who were randomised to receive the study drug or a sham control.
ZM-02 is an adeno-associated virus (AAV) vector-based gene therapy that delivers a light-sensitive protein gene to the eye via injection.
RP landscape remains barren
RP comprises a cluster of uncommon eye conditions that cause degeneration of the retina, the light-sensitive layer of tissue at the back of the eye, finally leading to a progressive loss of vision.
A report by GlobalData predicts that across the 16 major pharmaceutical markets (16MM: Australia, Brazil, Canada, China, France, Germany, India, Italy, Japan, Mexico, Russia, South Africa, South Korea, Spain, UK, and US), the number of diagnosed prevalent cases of RP will reach 1,088,269 in 2029.
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The RP treatment landscape is currently limited, with just one drug approved in the indication, Roche’s gene therapy Luxturna (voretigene neparvovec), which was approved in December 2017. While Roche holds the rights through its subsidiary Spark Therapeutics to market the drug in the US, Novartis holds the rights to market Luxturna outside of the US.
GlobalData predicts sales of Luxturna to reach $249m in 3031.
There is a limited a number of drugs, including betamethasone, inosine and troxerutin, which are used for symptomatic relief in a limited number of countries.
There are however several drugs in the pipeline, including Ocugen’s gene therapy OCU400 which has recently entered a Phase III trial, and Beacon Therapeutics’ gene therapy, laru-zova, which received regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) in January 2025.
Johnson and Johnson (J&J) was hoping to find a way into the RP space; however, its acquired drug, botaretigene sparoparvovec, failed to meet its primary endpoint in a Phase III trial. The big pharma acquired the drug as part of a $130m deal with MeiraGTx in late 2023.
According to GlobalData’s Pharmaceutical Intelligence Center, there are 34 candidates in trials for RP, with 22 of those in Phase II studies.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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