One-Time gene shot could free wilson patients from daily pills

NCT ID NCT06650319

Recruiting now Disease control Sponsor: Chaohui Yu Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Wilson disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

WILSON DISEASE

Contacts and locations

Locations

First Affiliated Hospital of Zhejiang University
RECRUITING

Hangzhou, Zhejiang, 312000, China

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