Gene therapy trial aims to fix copper buildup in Wilson's disease

NCT ID NCT07173933

Not yet recruiting Disease control Sponsor: GeneCradle Inc Source: ClinicalTrials.gov ↗

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.

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

Peking Union Medical College

Beijing, 100005, China

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