BRIX1 inhibitor and use thereof in resisting tumors

By using BRIX1 inhibitors, especially siRNA and microRNA, and utilizing exosome delivery systems to target the BRIX1 gene, the problems of insufficient cytotoxicity and p53 dependence of existing anti-tumor drugs on normal cells have been solved, achieving highly efficient and selective inhibition of tumor cells.

WO2026077291A1 Publication Date: 2026-04-16XINXIANG MEDICAL UNIV
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Patent Information

Application Number
PCT/CN2025/125154
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-10-09
Filing Date
2025-09-29
Publication Date
2026-04-16

AI Technical Summary

Technical Problem

Existing anti-tumor drugs are cytotoxic to normal cells when targeting nucleolar function, and they have the drawback of not being able to effectively activate or rely on the p53 pathway to inhibit tumor cells.

Method used

Develop BRIX1 inhibitors, including siRNA, microRNA and their precursors, to target the BRIX1 gene via exosome delivery systems, interfere with the nucleolar stress pathway, and activate or inhibit tumor cells in a p53-dependent manner.

Benefits of technology

It significantly inhibits tumor cell growth in vitro and in vivo, reduces the activity and expression of BRIX1 gene-encoded protein, especially effective against p53 wild-type tumors, enhances the effect of chemotherapy, and reduces toxicity to normal cells.

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Abstract

Disclosed in the present invention are a BRIX1 inhibitor and use thereof in resisting tumors. Specifically, disclosed in the present invention is that nucleolin BRIX1 is a key regulator of the homeostasis between ribosome biogenesis and p53 activation. The research results of the present invention show that the inhibition of BRIX1 can induce nucleolar stress, leading to activation of p53, thereby inhibiting tumor growth. In addition, the present invention constructs an engineered exosome whose surface is decorated with iRGD and which is loaded with BRIX1-specific siRNA and used for cancer therapy.
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