Encouraged by these pre-clinical results, AZD6738 is currently in the phase I trial in the treatment of MDS or CMML patients with at least one mutation in splicing factors (SF3B1, U2AF1, SRSF2, and ZRSR2), for safety and tolerability test (NCT03770429) [74,100]. The gene discussed is ZRSR2; the disease is myelodysplastic syndrome.