Gene therapy could free Wilson's patients from daily pills

NCT ID NCT07641140

Not yet recruiting Disease control Sponsor: Lingyi Biotech Co., Ltd. Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jul 02, 2026 · Updated 2 times

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.

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'S DISEASE

Contacts and locations

Locations

The First Affiliated Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310003, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.