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.
Disclaimer
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This is a summary of
the original study
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Shanghai General Hospital
RECRUITINGShanghai, Shanghai Municipality, 200080, China
Contact Phone: •••-•••-••••
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