Gene therapy trial aims to halt blindness from rare crystal disease
NCT ID NCT05694598
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a gene therapy called VGR-R01 for Bietti Crystalline Dystrophy, a rare inherited eye disease that leads to progressive vision loss. The study involves 12 adults who receive the therapy via injection into the eye. The main goal is to check safety and tolerability, while also measuring any changes in vision.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- VGR-R01 (a gene therapy that delivers a working copy of the CYP4V2 gene using a harmless virus)
- What this could lead to
- If it works, this could point toward a treatment that slows or stops vision loss in people with Bietti Crystalline Dystrophy.
- What could go wrong
- This is a very early, small Phase 1 trial with only 12 people, focused on safety. It may not show clear benefit, and gene therapies can have side effects like inflammation or immune reactions.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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Shanghai Vitalgen Biopharma Co.,Ltd.
Shanghai, Shanghai Municipality, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.