Base-edited Hematopoietic Stem and Progenitor Cells-National Institute of Allergy and Infectious Diseases (NIAID)-hyper-IgM syndrome

This is an investigational, autologous, ex vivo gene-edited cell therapy developed by the National Institute of Allergy and Infectious Diseases (NIAID) for the treatment of X-linked hyper-IgM syndrome (HIGM1). The therapy utilizes base editing technology to precisely correct mutations in the *CD40LG* gene, such as the c.658C>T (p.Q220X) mutation, within a patient's own hematopoietic stem and progenitor cells (HSPCs) and T cells. By repairing the genetic defect, the treatment aims to restore the expression of functional CD40 ligand (CD40L) on the surface of activated T cells, which is essential for B cell class switching and effective immune responses. The clinical protocol involves a single infusion of base-edited HSPCs following myeloid conditioning with busulfan and alemtuzumab, supplemented by a subsequent infusion of base-edited T cells to provide immediate immune support.

Other names

Base-edited hematopoietic stem and progenitor cellsBase-edited T lymphocyte cellsBase Editing Gene Therapy for CD40L-HyperIgM SyndromeBE-HSPCBE-T