Use of compound in preparing drug for treating hematologic malignancy
By using compounds with specific structures to act on the NUP214 gene, the problem of limited effectiveness of existing chemotherapy regimens in treating leukemia was solved, and the efficient inhibition of leukemia cells and the extension of mouse survival time were achieved.
WO2025189905A1 Publication Date: 2025-09-18CHONGQING MEDICAL UNIVERSITY
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Patent Information
- Application Number
- PCT/CN2024/141714
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-03-12
- Filing Date
- 2024-12-24
- Publication Date
- 2025-09-18
AI Technical Summary
Technical Problem
Existing chemotherapy regimens have limited therapeutic effects on leukemia, especially AML, and are prone to relapse. The lack of efficient drug interventions limits patients' survival rates.
Method used
A compound with a specific structure is provided for preparing a drug for treating blood tumors, especially leukemia, which acts on the NUP214 gene, degrades the protein expressed or fused to the gene, and inhibits the proliferation and development of leukemia cells.
Benefits of technology
The compound showed a highly effective inhibitory effect on multiple leukemia cell lines, significantly increased the mortality rate of leukemia cells, and prolonged the survival time of mice in a xenograft model, while having no significant effect on the expression of the NUP214 gene.
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Abstract
The present invention relates to use of a compound having a structure of formula (I) or a pharmaceutically acceptable salt thereof in preparing a drug for treating a hematologic malignancy.
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