Prime Medicine has dosed the first patient in a clinical trial of prime editing delivered directly into the body. The experimental treatment, PM577a, is designed to correct a genetic change linked to Wilson disease, an inherited condition that can cause excess copper to build up in the body.
Targeting the cause of Wilson disease
Wilson disease occurs when changes in the ATP7B gene impair the liver's ability to remove surplus copper. Current care commonly involves ongoing medication to manage copper levels. PM577a is designed to address one specific ATP7B mutation, H1069Q; it is not intended to cover every genetic variation that can cause the disease.
Prime editing is a gene-editing approach that can make targeted DNA changes without cutting both strands of the double helix. For this trial, the editing instructions are carried in lipid nanoparticles, which are delivered through an infusion and intended to reach liver cells.
Early research, with results still to come
In company-reported studies in genetically modified mice, the approach corrected the targeted change in some liver cells and reduced copper accumulation. Those findings do not establish that the treatment will work or have the same effect in people.
The Phase 1/2 trial will assess safety and monitor copper metabolism. Researchers will also examine whether participants can eventually reduce or stop their existing treatment. The first clinical results are expected in 2027; the therapy's effectiveness and long-term safety remain to be determined.
If clinical studies show that in-body gene editing can safely restore a missing function, it could help shape future treatments aimed at the genetic roots of some inherited diseases.
