期刊
CELL REPORTS PHYSICAL SCIENCE
卷 1, 期 12, 页码 -出版社
CELL PRESS
DOI: 10.1016/j.xcrp.2020.100277
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资金
- CIRM Therapeutic Translational Research Project entitled, A Splicing Modulator Targeting Cancer Stem Cells in Acute Myeloid Leukemia
While splice modulators have entered clinical trials, limited clinical efficacy in splicing factor mutation-driven malignancies, such as acute myeloid leukemia, has remained a challenge. There is a pressing unmet medical need for developing potent small molecule splice modulators for the treatment of a broad array of malignancies characterized by splicing deregulation. However, the inability to practically access gram-scale lead molecules with viable pharmacological properties continues to hinder their application. Here, we report a scalable approach to prepare 17S-FD-895, a potent in vivo active splice modulator. The strategy described herein not only provides material to enable clinical translation but also furthers lead validation by expanding the structure-activity relationships that guide splice modulation.
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