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Phase 1
Phase 1/2 Trial of Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-linked Chronic Granulomatous Disease
Background: Chronic granulomatous disease (CGD) is a rare immune disorder caused by a mutation in the CYBB gene. People with CGD have white blood cells that d…
Chronic Granulomatous Disease (CGD)X-Linked Chronic Granulomatous Disease
National Institute of Allergy and Infectious Diseases (NIAID)NCT06325709
Phase 1
A Phase 1/2 Open-Label, Single-Ascending-Dose Study of EN-374, a Helper-Dependent Adenoviral-Based Gene Therapy, in Participants With X-Linked Chronic Granulomatous Disease
The goal of this clinical trial is to evaluate the safety and potential efficacy of the EN-374 treatment regimen and identify a dose level for further evaluati…
X-Linked Chronic Granulomatous Disease
EnsomaNCT06876363