One-Time gene injection aims to halt rare eye disease

NCT ID NCT05832684

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a single injection of ZVS101e, a gene therapy that delivers a working copy of the CYP4V2 gene directly into the retina. It is for adults with Bietti's crystalline dystrophy, a rare inherited condition that leads to progressive vision loss. The trial will check whether the treatment is safe and can help preserve or improve eyesight.

What this could mean

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

Active substance

ZVS101e (a gene therapy carrying a working copy of the CYP4V2 gene)

What this could lead to

If successful, this could become the first treatment to slow or stop vision loss in people with Bietti's crystalline dystrophy.

What could go wrong

This is an early-phase trial with only 24 participants, so results may not apply to everyone. Gene therapy injections carry risks like inflammation or retinal damage.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

Bietti crystalline corneoretinal dystrophy

As listed by the trial registrant

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

Contacts and locations

Locations

  • Peking University Third Hospital

    Beijing, Beijing Municipality, 100191, China

  • Tianjin Medical University Eye Hospital

    Tianjin, Tianjin Municipality, 300392, China

  • West China Hospital, Sichuan University

    Chengdu, Sichuan, China