Molecularly driven agents such as IDH1/2 inhibitors, venetoclax, magrolimab, and APR-246 for MDS, as well as tagraxofusp, tipifarnib, and lenzilumab for CMML are being evaluated in various stages of clinical trials but more data are needed prior to their use in routine clinical practice. The gene discussed is IDH1; the disease is myelodysplastic syndrome.