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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for BIETTI CRYSTALLINE CORNEORETINAL DYSTROPHY are added.
By submitting, you agree to our Terms of use
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
-
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a single injection restore sight in a rare genetic eye disease?
- One-Time gene injection aims to halt rare blindness
- Gene therapy takes on rare eye disease in Second-Eye trial
- Gene therapy injection aims to halt blindness from rare eye disorder
- Gene therapy trial aims to halt blindness from rare crystal disease
- Experimental gene therapy offers hope for rare blindness condition