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A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Modified Ex Vivo Using Prime Editing (PM359) in Participants With Autosomal Recessive Chronic Granulomatous Disease Due to Mutations in the NCF1 Gene

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Trial Profile

A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Modified Ex Vivo Using Prime Editing (PM359) in Participants With Autosomal Recessive Chronic Granulomatous Disease Due to Mutations in the NCF1 Gene

Status: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 30 May 2025

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At a glance

  • Drugs PM 359 (Primary)
  • Indications Chronic granulomatous disease
  • Focus Adverse reactions; First in man; Proof of concept; Therapeutic Use
  • Sponsors Prime Medicine

Most Recent Events

  • 25 May 2025 Status changed from recruiting to active, no longer recruiting.
  • 19 May 2025 Positive initial data from the first patient dosed in its ongoing Phase 1/2 clinical study of PM359 in Chronic Granulomatous Disease (CGD). presented in the Prime Medicine Media Release.
  • 28 Feb 2025 According to a Prime Medicine media release, company will Announce initial clinical data from Cohort 1 in Phase 1/2 trial of PM359 for p47phox CGD in 2025.

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