BE-HSPC IL2RG

BE-HSPC IL2RG is an investigational autologous gene-edited cell therapy developed by the National Institute of Allergy and Infectious Diseases (NIAID) for the treatment of X-linked severe combined immunodeficiency (X-SCID). The therapy utilizes base editing technology—a precise form of CRISPR-based genome editing—to repair mutations in the interleukin 2 receptor gamma (IL2RG) gene within a patient's own hematopoietic stem and progenitor cells (HSPCs). The IL2RG gene encodes the common gamma chain (γc), a protein essential for the signaling of multiple interleukins (IL-2, IL-4, IL-7, IL-9, IL-15, and IL-21) that drive the development and function of T cells, B cells, and natural killer (NK) cells. By correcting the genetic defect ex vivo and re-infusing the cells into the patient following a busulfan conditioning regimen, the therapy aims to restore long-term immune competence and lymphocyte maturation.

Other names

Base-edited hematopoietic stem and progenitor cellsBase-edited autologous CD34 plus hematopoietic stem and progenitor cell product