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|Molecular Profile||Indication/Tumor Type||Response Type||Therapy Name||Approval Status||Evidence Type||Efficacy Evidence||References|
|SF3B1 K700E||acute myeloid leukemia||sensitive||H3B-8800||Preclinical - Pdx||Actionable||In a preclinical study, H3B-8800 inhibited aberrant splicing and tumor growth in a cell line xenograft and a patient-derived xenograft (PDX) model of acute myeloid leukemia harboring SF3B1 K700E (PMID: 29457796).||29457796|
|SF3B1 K700E||acute myeloid leukemia||sensitive||H3B-8800||Preclinical - Cell culture||Actionable||In a preclinical study, H3B-8800 preferentially inhibited growth of mouse acute myeloid leukemia cells harboring SF3B1 K700E compared to SF3B1 wild-type cells in culture (Blood 2016 128:966).||detail...|
|PubMed Id||Reference Title||Details|
|H3B-8800, an Orally Bioavailable Modulator of the SF3b Complex, Shows Efficacy in Spliceosome-Mutant Myeloid Malignancies||Full reference...|
|(29457796)||H3B-8800, an orally available small-molecule splicing modulator, induces lethality in spliceosome-mutant cancers.||Full reference...|