In AML, EAPB0503 exclusively targeted mutated NPM1, inducing selective growth arrest and apoptosis in NPM1c AML cells in vitro [32] and NPM1c AML patient blasts ex vivo [29], along with a remarkable therapeutic efficacy in treated xenograft mice with mutated NPM1 [29,32]. The gene discussed is NPM1; the disease is acute myeloid leukemia.