Gene therapy injection aims to save sight in rare eye disease

NCT ID NCT07282457

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tests a single injection of ZM-02, a gene therapy, in 21 people aged 6 to 60 with advanced retinitis pigmentosa, a genetic condition that causes progressive vision loss. Participants are randomly assigned to receive either a low or high dose of ZM-02 or a sham injection. The study primarily checks safety and tolerability, while also measuring changes in visual acuity, mobility in low light, and quality of life over time.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
ZM-02 (gene therapy injection)
What this could lead to
If successful, this could point toward a treatment that slows or partially reverses vision loss in retinitis pigmentosa.
What could go wrong
This is an early phase 1/2 trial with only 21 people, so results may not apply widely. Gene therapies can have side effects like inflammation, and the sham control means some may not receive the active treatment.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

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