Experimental gene injection aims to save sight in rare eye disease
NCT ID NCT06066008
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tested a gene therapy called ZM-01 in 2 boys aged 3 to 18 with X-linked retinoschisis, a rare genetic eye condition that causes vision loss. Each boy received an injection of the therapy into one eye. The main goal was to check safety, but researchers also measured changes in vision. Because the study is very small and early, it cannot prove the treatment works.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- ZM-01 (rAAV-hRS1) gene therapy injection
- What this could lead to
- If it works, this could point toward a treatment that slows vision loss in X-linked retinoschisis.
- What could go wrong
- This is a very early, tiny trial with only 2 participants. It mainly checks safety, not effectiveness, and may not lead to a working 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.
Contacts and locations
Locations
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Wuhan University Renmin Hospital affiliated with Hanchuan Hospital
Xiaogan, Hubei, China
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Other studies related to the condition(s) this trial covers.