BIOMARKERS FOR MYELODYSPLASTIC SYNDROME (MDS) AND METHODS OF USING THE SAME

    公开(公告)号:WO2022098712A1

    公开(公告)日:2022-05-12

    申请号:PCT/US2021/057839

    申请日:2021-11-03

    Abstract: The present disclosure relates to the treatment of transfusion dependence in myelodysplastic syndrome (MDS). The present disclosure relates to methods of using novel biomarkers to treat transfusion dependence in an MDS patient in need thereof. The present disclosure also relates to methods of identifying MDS patients suitable for treatment with a splicing modulator and/or predicting or monitoring treatment efficacy in an MDS patient. In some embodiments, the methods disclosed herein comprise determining at least the ratio of aberrant junction to canonical junction TMEM14C transcripts (TMEM14C AJ/CJ ratio) in the patient. In some embodiments, the methods disclosed herein comprise administering a therapeutically effective amount of a splicing modulator (e.g., Compound 1) based on the patient's TMEM14C AJ/CJ ratio. Therapeutic uses and compositions are also disclosed.

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