Zhongmou Therapeutics announced that first-in-human (FIH) clinical data from its MOON trial evaluating ZM-02 were featured in an invited oral presentation at the Advanced Therapies Congress (ATC-2025), held November 18–19 in Philadelphia.

Zhenghong Gao, PhD, Chief Strategy Officer of Zhongmou, delivered the presentation, “Restoring Vision by Optogenetics,” highlighting topline FIH data from ZM-02 and leading a panel discussion on “Innovations in Novel Capsids for Gene Therapies.”

“ATC provides a critical scientific forum for advancing next-generation medicines,” said Dr. Gao. “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.”

MOON Trial (NCT06292650): Key Findings from the ZM-02 First-in-Human Study

The MOON trial is the first randomized clinical study evaluating a mutation-independent, optogenetic gene therapy for advanced retinitis pigmentosa (RP).

Safety

  • Robust safety profile with no drug-related serious adverse events (SAEs) and no dose-limiting toxicities (DLTs) across all dose cohorts.

Visual Acuity & Functional Efficacy

  • 83% of treated patients achieved ≥0.3 LogMAR BCVA improvement at Week 36

  • Mean visual acuity gain reached 0.59 LogMAR—equivalent to approximately 30 ETDRS letters

  • Patients demonstrated marked improvements in multi-luminance mobility testing, including low-light navigation

Real-World Functional Gains

  • Participants showed rapid recovery of daily navigation ability

  • The trial documented the first restoration of color vision in advanced RP using an optogenetic approach

  • Some patients successfully returned to activities such as cycling without external visual aids

Durability

Although the ATC presentation shared Week-36 results, all participants have now completed 52-week follow-up, confirming sustained improvements in visual acuity, mobility, and color perception after a single intravitreal injection of ZM-02.

“Advanced RP is a disease where gene-specific therapies typically fail due to near-complete photoreceptor loss,” said Yin Shen, MD, PhD, Founder and CEO of Zhongmou. “ZM-02 directly addresses this unmet need by bypassing degenerated photoreceptors and reactivating residual inner retinal circuits. At ATC 2025, we presented compelling evidence of patients transitioning from complete blindness to functional vision—regaining navigation capability in daily environments and even safely returning to activities such as cycling. These data represent a foundational step toward redefining what is clinically possible in late-stage RP.”

According to Zhongmou, an IND filing for a phase 1/2 trial remains on track. The FDA granted ZM-02 Orphan Drug Designation in October 2024.