Gene therapy injection aims to save sight in rare genetic disease
NCT ID NCT07269665
First seen Jan 07, 2026 · Last updated Jun 22, 2026 · Updated 30 times
Summary
This early-stage trial tests a single injection of AXV-101 gene therapy into one eye of 12 children and teens (ages 4-17) with Bardet-Biedl syndrome type 1, a rare genetic condition that causes progressive vision loss. The main goal is to see if the treatment is safe and tolerable over 5 years. Researchers will compare the treated eye to the untreated eye to look for any signs of benefit.
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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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Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Moorfields Eye Hospital
London, EC1V 2PD, United Kingdom
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
AXV-101 (gene therapy injected into the eye)
What this could lead to
If it works, this could slow or stop vision loss in people with BBS1, potentially preserving sight for years.
What could go wrong
This is a very early, first-in-human trial with only 12 people. It is designed mainly to check safety, not effectiveness. Gene therapy in the eye carries risks like inflammation or infection.
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.