Can a single infusion rewrite the genetic code behind wilson disease?
NCT ID NCT07748403
First seen Aug 05, 2026 · Last updated Aug 12, 2026 · Updated 3 times
Summary
This trial tests an experimental therapy called PM577a, which uses prime editing to correct a common genetic mutation that causes Wilson disease. The treatment is given as a single intravenous infusion and aims to restore the liver's ability to remove excess copper. The study includes adults and adolescents with Wilson disease who have at least one copy of the p.H1069Q mutation. Researchers will monitor safety, how the body responds, and whether copper metabolism improves.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- PM577a, an investigational prime editing therapy designed to correct the p.H1069Q mutation in the ATP7B gene
- What this could lead to
- If successful, this could lead to a one-time treatment that corrects the genetic cause of Wilson disease, potentially allowing patients to stop lifelong copper-removing medications.
- What could go wrong
- This is an early-stage, first-in-human trial, so safety and effectiveness are unproven. The editing may not work in all cells, and there are risks of immune reactions or unintended genetic changes.
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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
Locations
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New Zealand Clinical Research (NZCR)
Grafton, Auckland, 1010, New Zealand
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