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Anti-CD117 CAR T-cells are chimeric antigen receptor (CAR) T cell therapies genetically engineered to express a receptor targeting CD117 (also known as KIT), a type III receptor tyrosine kinase highly expressed on hematopoietic stem and progenitor cells (HSPCs) and most acute myeloid leukemia (AML) cells. Their mechanism is to specifically recognize and eliminate CD117+ malignant and healthy HSPCs, enabling deep myeloablation prior to hematopoietic stem cell transplantation and efficiently depleting leukemic stem cells. These CAR T-cells have demonstrated potent activity *in vitro* and *in vivo* for the treatment of AML and advanced systemic mastocytosis, and show potential as conditioning regimens prior to allogeneic transplant. Safety switch mechanisms (e.g., inducible Caspase 9) have been utilized to provide controlled CAR T-cell ablation in vivo. The therapy is under preclinical and early clinical development; key development and research published as of 2023.
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