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Anti-NPM1c CAR-NK cells are a genetically engineered cell therapy product consisting of cytokine-induced memory-like natural killer (CIML NK) cells modified to express a chimeric antigen receptor (CAR) that specifically targets a neoepitope derived from the mutated NPM1 protein ("NPM1c") presented in complex with HLA-A*0201. This mutation is found in about 30% of all adult acute myeloid leukemia (AML) cases and is associated with poor prognosis. The CAR confers antigen-specific cytotoxic activity to the NK cells, significantly enhancing their ability to recognize and destroy AML cells carrying NPM1c mutations, while retaining their intrinsic, non-antigen-dependent antileukemia activity. The engineered product demonstrates potent antileukemia activity in vitro and in preclinical models, showing specificity for AML cells carrying the NPM1c mutation and minimal off-target effects[1][7].
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