One-Time gene injection aims to halt rare blindness

NCT ID NCT07653971

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

Summary

This trial tests a single injection of a gene therapy called ZVS101e for people with Bietti crystalline dystrophy, a rare inherited eye disease that leads to progressive vision loss. The therapy uses a harmless virus to deliver a working copy of the CYP4V2 gene to cells in the eye. Participants receive one injection in the study eye and are followed for 4 weeks to check safety and early signs of effectiveness.

What this could mean

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

Active substance
ZVS101e (a gene therapy injection using a harmless virus to deliver a working copy of the CYP4V2 gene)
What this could lead to
If successful, this could provide a treatment option for people with Bietti crystalline dystrophy, potentially slowing vision loss or improving sight.
What could go wrong
This is an early-stage trial with a small number of participants, so results may not apply to everyone. Gene therapy carries risks like inflammation or retinal damage.

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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

  • Boao Super Hospital

    Qionghai, Hainan, China

  • Hainan Boao Ophthalmology Center, Zhongshan Ophthalmic Center, Sun Yat-sen University

    Qionghai, Hainan, China

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