China’s Zhongmou Therapeutics announced Nov. 27 that the US FDA had signed off on its investigational new drug (IND) application, allowing the company to proceed with a Phase I/II trial of ZM-02, the company’s optogenetic candidate for patients with advanced retinitis pigmentosa (RP).

The mutation-agnostic gene therapy candidate gained orphan drug status from the FDA in October 2024.

Optogenetic therapy uses gene delivery to introduce light-sensitive proteins called opsins into surviving retinal neurons, enabling them to respond to light after native photoreceptors are lost.

ZM-02 is delivered in a single intravitreal injection. It expresses a novel engineered photosensitive protein, PsCatCh2.0e, in inner retinal neurons to restore light responsiveness in blind eyes.

Endpoints News reported that Zhongmou tested its treatment in an investigator-initiated trial, which has become common in China. The company recruited 12 patients with advanced retinal degeneration caused by mutations in several different genes. Two patients got a low dose injected into one eye, seven patients got a high dose, and three got a sham injection.

All nine treated patients had some improvement in vision, and seven saw clinically significant improvement, Zhongmou told Endpoints. On average, treated patients improved by around 30 letters on an eye chart. Patients in the placebo group didn’t improve.

Zhongmou also said that patients went from seeing in almost entirely grayscale to being able to perceive colors. The company believes this result may be unique among ocular gene therapy trials.

The Phase I/II PRISM trial will enroll adults and children with retinitis pigmentosa in the US and China. Key assessments will include safety endpoints, ophthalmic exams, and functional vision testing, including a multi-luminance mobility test, multi-luminance chessboard test, and multi-luminance shape discrimination. Follow-up will occur at 52 weeks and beyond, Zhongmou said.

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