Recent advances in the understanding of the biology of MDS/CMML and AML led to the development of novel targeted and untargeted agents (e.g., IDH inhibitors, FLT3 inhibitors, splicing modulators, novel DNMTi, anti-CD33 antibodies) [15,16,17,18,19,20]. The gene discussed is IDH1; the disease is acute myeloid leukemia.