FDA clears IND for PM577a, an in vivo prime editor for H1069Q Wilson disease

Prime Medicine/PM577a (Wilson disease)

The FDA cleared Prime Medicine to test PM577a in people. It is a prime editor, a gene-editing tool that rewrites a short stretch of DNA without cutting both strands, delivered to the liver in lipid nanoparticles in a single infusion. It corrects the H1069Q mutation in ATP7B, the gene that clears copper from the body; when it fails, copper builds up in the liver and brain (Wilson disease).

• H1069Q accounts for about 30–50% of Wilson disease variants in the US and Europe; the disease affects about 1 in 30,000 people.
• With an earlier New Zealand clearance, this makes a global Phase 1/2: open-label, rising doses, adults and adolescents who are stable on standard treatment.
• Trial start planned for the second half of 2026; first clinical data expected in 2027.
• Today's treatment is lifelong copper-removing drugs; a one-time liver correction would aim to replace them.