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A Phase 1/2 Clinical Study to Evaluate Safety, Tolerability, Biological Activity, and Initial Efficacy of Prime Editing (PM577a) for the Treatment of Wilson Disease (WD) in Adult and Adolescent Participants With at Least One Allele Harboring the p.H1069Q Mutation in ATP7B

Trial Profile

A Phase 1/2 Clinical Study to Evaluate Safety, Tolerability, Biological Activity, and Initial Efficacy of Prime Editing (PM577a) for the Treatment of Wilson Disease (WD) in Adult and Adolescent Participants With at Least One Allele Harboring the p.H1069Q Mutation in ATP7B

Status: Not yet recruiting
Phase of Trial: Phase I/II

Latest Information Update: 13 Aug 2026

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At a glance

  • Drugs PM 577 (Primary)
  • Indications Hepatolenticular degeneration
  • Focus Adverse reactions; First in man
  • Sponsors Prime Medicine

Most Recent Events

  • 06 Aug 2026 According to Prime Medicine media release, company expects to initiate global phase 1/2 study of PM577a in the second half of 2026, with initial clinical data anticipated in 2027.
  • 05 Aug 2026 Status changed from planning to not yet recruiting.
  • 18 Jun 2026 According to Prime Medicine media release, the company plans to initiate this study in 2H of 2026.

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