The ASO BP1001 targeting the translation initiation site of the growth factor receptor-bound protein 2 (Grb2) has been tested in acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS) and showed a good safety profile and evidence of antileukemic activity in a phase I study [13]. This evidence concerns the gene GRB2 and myelodysplastic syndrome.