A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

NCTID	NCT06559176 (View at clinicaltrials.gov)
Description	This is an open-label, single-arm, multicenter Phase 1/2 study evaluating the safety and efficacy of gene therapy by transplantation of Prime Edited autologous CD34+ stem cells modified ex vivo (PM359) in participants with autosomal recessive Chronic Granulomatous Disease (CGD) caused by mutations in the NCF1 (Neutrophil Cytosolic Factor 1) gene. (Show More)
Development Status	Active
Indication	Chronic Granulomatous Disease
Disease Ontology Term	DOID:0070192
Compound Name	PM359
Sponsor	Prime Medicine, Inc.
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	12
Results Posted	Not Available

No.of Trial Sites	5
Locations	Canada,United States,United Kingdom

Has US IND	True
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Initial data from Phase 1/2 clinical trial of PM359 for p47phox CGD expected in 2025