DOT1L inhibitors (Pinometostat/EPZ-5676), along with SNDX-5613 and KO-539 (targeting MENIN, a protein involved in MLL::FP recruitment), are being used in clinical trials on MLL-r AML, as well as other subtypes, including NPM1-mutated AML [38,109,110,111]. The gene discussed is NPM1; the disease is acute myeloid leukemia.