Gene therapy trial aims to fix copper buildup in Wilson's disease
NCT ID NCT07173933
First seen Jun 26, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This early-stage trial tests a gene therapy called GC310 for Wilson's disease, a genetic condition that causes copper buildup in the body. Researchers will give a single dose to 15 adults and monitor them for a year to check safety and whether it improves copper levels. The goal is to see if this approach can help control the disease.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
GC310 gene therapy (AAV5 vector delivering a functional ATP7B gene)
What this could lead to
If successful, this could provide a one-time gene therapy that helps control Wilson's disease by restoring copper processing, potentially reducing the need for daily medications.
What could go wrong
This is an early phase I/II trial with only 15 participants, so safety and effectiveness are not yet proven. Gene therapies can have side effects like immune reactions, and long-term benefits are unknown.
Disclaimer
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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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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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Peking Union Medical College
Beijing, 100005, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
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