First human trial launches for gene therapy targeting rare blindness disorder
NCT ID NCT07269665
First seen Jan 07, 2026 · Last updated May 15, 2026 · Updated 23 times
Summary
This early-stage trial tests a single dose of AXV-101, a gene therapy, in 12 children aged 4 to 17 with Bardet-Biedl syndrome type 1 (BBS1) who have vision loss. The main goal is to see if the treatment is safe and tolerable, and to find the right dose. Participants will have eye exams, blood and tear tests, and quality-of-life surveys over one year, with safety monitored for up to five years.
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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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Study contacts
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Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Moorfields Eye Hospital
London, EC1V 2PD, United Kingdom
Conditions
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