Base-edited hematopoietic stem and progenitor cells
Sponsors
National Institute of Allergy and Infectious Diseases (NIAID)
Conditions
CD40L-HyperIgM SyndromeChronic Granulomatous Disease (CGD)HypogammaglobulinemiaImmunodeficiencyMyelokathexisWHIMWartsX-Linked Chronic Granulomatous Disease
Phase 1
Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease
RecruitingNCT06325709
Start: 2024-04-17End: 2032-12-31Target: 15Updated: 2026-08-13
Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
Enrolling by invitationNCT06851767
Start: 2025-05-09End: 2034-12-31Target: 18Updated: 2026-09-03
Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study
RecruitingNCT06959771
Start: 2025-07-16End: 2027-10-28Target: 1Updated: 2026-07-31
Base-Edited Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of CXCR4-WHIM
Not yet recruitingNCT07775313
Start: 2026-09-16End: 2033-12-31Target: 10Updated: 2026-09-11