One-Time gene injection aims to halt rare blindness

NCT ID NCT07653971

First seen Jun 27, 2026 · 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 — 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.

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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Boao Super Hospital

    Qionghai, Hainan, China

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

    Qionghai, Hainan, China