Mutant SF3B1 can be targeted by the macrocyclic lactone Pladienolide B. Its derivatives, including E7107 and H3B-8800, have demonstrated effectiveness against various mutant splicing factors, and H3B-8800 is currently undergoing a first-in-human Phase I clinical trial for the treatment of chronic myelomonocytic leukaemia, AML and MDS. The gene discussed is SF3B1; the disease is myelodysplastic syndrome.