Clinical TrialJun 18, 2026, 08:01 AM
Prime Medicine gets first clinical authorization for in vivo Prime Editing
AI Summary
Prime Medicine, Inc. announced that the New Zealand Medicines and Medical Devices Safety Authority (Medsafe) has cleared its Clinical Trial Application (CTA) for PM577a, an investigational Prime Editor for Wilson Disease. This represents the company's first clinical authorization for an in vivo Prime Editing therapy. Prime Medicine expects to initiate a global Phase 1/2 clinical trial in the second half of 2026, with initial clinical data anticipated in 2027. PM577a targets the H1069Q mutation, the most prevalent Wilson Disease-causing allele in North America and Europe, with plans for additional products to address other pathogenic variants globally.
Key Highlights
- New Zealand Medsafe cleared CTA for PM577a, an investigational Prime Editor for Wilson Disease.
- This marks Prime Medicine's first clinical authorization for an in vivo Prime Editing therapy.
- Phase 1/2 clinical trial expected to start in H2 2026; initial data expected in 2027.
- PM577a targets the H1069Q mutation, accounting for 30-50% of WD variants in US/Europe.
- A follow-on candidate targeting the R778L mutation (common in East Asia) is in preclinical development.
- Wilson Disease is a rare genetic disorder affecting an estimated 1 in 30,000 individuals globally.
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