The identification of reoccurring genetic alterations has spurred the development of targeted therapies for AML and by now multiple drugs have entered the clinic: the tyrosine kinase inhibitors midostaurin and gilteritinib have proven efficacious in the treatment of patients harboring FLT3 mutations and mutation-specific inhibitors of IDH1 and IDH2 have shown promising activity in patients with relapsed AML. The gene discussed is IDH2; the disease is acute myeloid leukemia.