Gene therapy injection aims to restore sight in rare eye disease

NCT ID NCT06743646

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

Summary

This study tests a single injection of ZVS101e gene therapy for people with Bietti's crystalline dystrophy, a rare inherited condition that leads to progressive vision loss. The therapy delivers a working copy of the CYP4V2 gene directly to the retina. Researchers will measure whether treated eyes gain at least 15 letters on an eye chart compared to untreated eyes.

What this could mean

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

Active substance

ZVS101e (gene therapy)

What this could lead to

If successful, this could become the first approved treatment for Bietti's crystalline dystrophy, potentially slowing vision loss or improving sight.

What could go wrong

This is an early-stage trial with only 62 participants, so results may not apply to everyone. Gene therapy carries risks like inflammation or uncertain long-term effects.

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

  • Eye Hospital, Wenzhou Medical University

    Wenzhou, Zhejiang, China

  • Henan Provincial People's Hospital

    Zhengzhou, Henan, China

  • Peking University Third Hospital

    Beijing, Beijing Municipality, China

  • The First Affiliated Hospital of Harbin Medical University

    Haerbin, Heilongjiang, China

  • Tianjin Medical University Eye Hospital

    Tianjin, Tianjin Municipality, China

  • West China Hospital of Sichuan University

    Chengdu, Sichuan, China

  • Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

    Shanghai, Shanghai Municipality, China

  • Zhongshan Ophthalmic Center, Sun Yat-sen University

    Guangzhou, Guangdong, China