Gene therapy injection aims to save sight in rare eye disease
NCT ID NCT07002398
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial tests a new gene therapy called VG801 for Stargardt disease, a genetic condition that causes vision loss. Fifteen people aged 6 and older will receive a single injection under the retina. The main goal is to check safety, but researchers will also measure 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
- VG801 (a gene therapy drug injected under the retina)
- What this could lead to
- If it works, this could point toward a treatment that slows or stops vision loss in people with Stargardt disease.
- What could go wrong
- This is a very early, first-in-human trial with only 15 participants. It may not work, and there are risks from the injection and gene therapy itself.
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 RETINAL DYSTROPHY DUE TO BIALLELIC ABCA4 MUTATIONS 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
-
Shanghai General Hospital
RECRUITINGShanghai, Shanghai Municipality, 200080, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.