Prime Medicine doses first patient in Phase 1/2 trial of PM577a for H1069Q-mutated Wilson disease
PRME•Prime Medicine dosed the first patient in a global Phase 1/2 trial of PM577a, a one-time gene-editing treatment for H1069Q-mutated Wilson disease. Initial clinical data are expected in 2027, and the FDA granted PM577 Rare Pediatric Disease designation.
1. Trial begins
Prime Medicine dosed the first patient in a global Phase 1/2 trial of PM577a for H1069Q-mutated Wilson disease. The first-in-human study aims to assess safety and early signs of benefit from the one-time gene-editing treatment; initial clinical data are expected in 2027.
2. FDA designation
The FDA granted PM577 Rare Pediatric Disease designation, supporting the program’s regulatory strategy in a childhood-onset condition.
