Prime Medicine doses first patient with in vivo prime editing for Wilson disease

Prime Medicine has dosed the first Wilson disease patient with PM577a, the first known use of prime editing inside a living person. Lipid nanoparticles deliver the editing machinery to liver cells to correct the H1069Q mutation in ATP7B, with initial clinical data expected in 2027.

• PM577a targets the H1069Q mutation in ATP7B via intravenous infusion
• Lipid nanoparticles deliver the editors to hepatocytes without cell extraction
• The Phase 1/2 trial is cleared by the FDA and New Zealand's Medsafe
• Only dosing feasibility is confirmed; safety and efficacy data are due in 2027