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Clinical trial

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

NCT06559176 is registered in ClinicalTrials.gov with Prime Medicine, Inc. as lead sponsor.

NCT NCT06559176
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Registry record

As filed in ClinicalTrials.gov.

Title
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
Lead sponsor
Prime Medicine, Inc.
Phase
Phase 1/2
Overall status
Enrolling by invitation
Start date
October 17, 2024
Completion date
February 28, 2030
Enrollment
12 (estimated)
Sites
5

Conditions

As the registry lists them; a condition with its own page is linked.

chronic granulomatous disease, granulomatous disease, chronic