Prime Medicine doses first patient in global Phase 1/2 trial of PM577a for Wilson disease
Prime Medicine announced that the first patient was dosed in its worldwide Phase 1/2 study of PM577a, an in‑vivo Prime Editing gene therapy targeting the H1069Q mutation that causes Wilson disease. The U.S. FDA granted Rare Pediatric Disease designation to the therapy, which could make the company eligible for a Priority Review Voucher. The open‑label trial will assess safety, tolerability and early efficacy in adults and adolescents, with initial data expected in 2027.
Why it matters
The company says the Rare Pediatric Disease designation highlights the unmet need and may enable a priority review voucher, potentially accelerating future approvals. Investors may view the start of the first in‑vivo Prime Editing trial as a milestone that could de‑risk the program and support the valuation of Prime Medicine.
Key facts
- 1Prime Medicine dosed the first patient in the global Phase 1/2 trial of PM577a on Oct. 5, 2026. globenewswire.com
- 2The trial targets the H1069Q mutation, the most prevalent Wilson disease‑causing allele in North America and Europe. globenewswire.com
- 3The U.S. FDA granted Rare Pediatric Disease designation to PM577 for Wilson disease. globenewswire.com
- 4Initial clinical data from the trial are expected in 2027. globenewswire.com
- 5The Phase 1/2 study is open‑label, global, and will evaluate safety, tolerability and preliminary efficacy in adults and adolescents with at least one H1069Q allele. investing.com
- 6Efficacy measures may include copper efflux by 64Cu PET, serum ceruloplasmin, non‑ceruloplasmin‑bound copper and 24‑hour urinary copper excretion. investing.com
Summary written by AlphAI from 2 of 2 sources. Not investment advice. Figures are as stated by the linked sources.