One-Time gene shot could free wilson patients from daily pills
NCT ID NCT06650319
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests a single dose of LY-M003, a gene therapy that delivers a working copy of the ATP7B gene to liver cells. The goal is to see if it is safe and can reduce the need for standard Wilson disease medications. The study enrolls 18 adults and children with confirmed Wilson disease.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
LY-M003 (gene therapy using a harmless virus to deliver a working ATP7B gene to liver cells)
What this could lead to
If successful, this could reduce or eliminate the need for daily medication to manage copper buildup in Wilson disease patients.
What could go wrong
This is a very early, small trial (18 people) focused on safety. Gene therapy can have side effects, and it may not work as hoped. Long-term benefits are uncertain.
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: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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First Affiliated Hospital of Zhejiang University
RECRUITINGHangzhou, Zhejiang, 312000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••