GlobeNewswire·Jun 18, 2026, 8:00 AM ET

Prime Medicine Announces New Zealand Clearance of Clinical Trial Application for PM577a in H1069Q-mutated Wilson Disease

New Zealand's Medsafe cleared Prime Medicine's Clinical Trial Application for PM577a, marking the first in vivo Prime Editing therapy authorization for the company.
PM577a targets the H1069Q mutation in the ATP7B gene, the most prevalent Wilson Disease-causing allele in North America & Europe.
The global Phase 1/2 clinical trial is expected to initiate in the second half of 2026, with initial clinical data anticipated in 2027.
The trial will be an open-label, first-in-human study evaluating safety, tolerability, & efficacy in adults & adolescents, initially enrolling patients stable on standard-of-care therapy.
Biological activity assessments will include 64 Cu PET copper efflux, serum ceruloplasmin, non-ceruloplasmin bound copper, 24-hour urinary copper excretion, & hepatic copper biopsy.
PM577a is an LNP-formulated Prime Editor designed to correct pathogenic ATP7B mutations via a single intravenous infusion.
A shared LNP delivery platform may enable rapid expansion into additional Wilson Disease populations, with a follow-on candidate targeting the R778L mutation in East Asian populations in pre-clinical development.
Wilson Disease is a rare genetic disorder affecting 1 in 30,000 individuals globally, currently lacking curative options beyond liver transplantation.
Prime Medicine is utilizing its proprietary Prime Editing platform to develop one-time curative therapies across liver, lung, & immunology/oncology programs.