Over the past 10 years, tremendous progress has been made in the development of approaches for treating AML, such as hematopoietic stem cell transplantation; inhibitors targeting MCL-1, IDH2, and NPM1/FLT3-ITD mutations, epigenetic agents, antibody-based treatments, and cellular therapies. The gene discussed is FLT3; the disease is acute myeloid leukemia.