The therapeutic candidate has also received a Rare Pediatric Disease designation from the FDA. The trial focuses on the safety and efficacy of using in vivo Prime Editing to treat the specific genetic mutation associated with the disease.
Wednesday, October 21, 2026
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Tech15 days ago
A global Phase 1/2 clinical trial of PM577a for H1069Q-mutated Wilson disease has commenced with the dosing of its first patient. This milestone represents the first clinical evaluation of an in vivo Prime Editing therapy developed by Prime Medicine, marking the shift of its editing technology into human trials.

The therapeutic candidate has also received a Rare Pediatric Disease designation from the FDA. The trial focuses on the safety and efficacy of using in vivo Prime Editing to treat the specific genetic mutation associated with the disease.
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