Many targeted agents such as isocitrate dehydrogenase (IDH) Inhibitors (IDH1, ivosidenib; IDH2, enasidenib), hedgehog (Hh) inhibitor (glasdegib), and BCL2 inhibitor (venetoclax) in combination therapy have been evaluated and showed encouraging results in relapsed/refractory (R/R) AML or in AML/MDS patients ineligible for intensive chemotherapy (111–117). The gene discussed is IDH1; the disease is myelodysplastic syndrome.