Gene therapy could free Wilson's patients from daily pills
NCT ID NCT07641140
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a single-dose gene therapy called LY-M003 in 18 adults with Wilson's disease, a genetic condition that causes copper buildup. The goal is to see if it's safe and can reduce or eliminate the need for standard daily medications. Participants receive one intravenous infusion and are monitored for side effects and treatment response.
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 (a gene therapy given as a single intravenous infusion)
What this could lead to
If successful, this could allow people with Wilson's disease to reduce or stop their daily medications, potentially controlling the disease with a one-time treatment.
What could go wrong
This is an early, small trial (18 people) testing safety and dosing. Gene therapies can have serious side effects, and it's unknown if the effect will last or if lifelong medication will still be needed.
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
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