Title:
SIRNA OR SALT THEREOF AND MEDICAMENT FOR INHIBITING EXPRESSION OF TMPRSS6 GENE, AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2024/060649
Kind Code:
A1
Abstract:
The present invention provides an siRNA or a pharmaceutically acceptable salt for inhibiting the expression of TMPRSS6 in a human cell, and a proper modification is made to the siRNA to improve the silencing capability against a target and to reduce off-target activity. The present invention also provides a biological preparation or a pharmaceutical preparation containing the siRNA and used for inhibiting the expression of TMPRSS6. According to the present invention, by means of a series of in-vitro and in-vivo tests, an siRNA sequence with good biological activity in inhibiting the expression of TMPRSS6 is obtained, and the activity is higher than that of TMPRSS6-HCM-9 with the fastest clinical progress, such that the present invention is expected to be clinically applied to the treatment of a disease related to abnormal expression of TMPRSS6, or a disease associated with iron excess load or iron overload, thus having good application prospects.
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Inventors:
LIU XINJIAN (CN)
FAN FUSHUN (CN)
QIAN CHANGGENG (CN)
FAN FUSHUN (CN)
QIAN CHANGGENG (CN)
Application Number:
PCT/CN2023/095104
Publication Date:
March 28, 2024
Filing Date:
May 18, 2023
Export Citation:
Assignee:
BEBETTER MED INC (CN)
International Classes:
C12N15/113; A61K31/713; A61P7/00; A61P7/06
Foreign References:
| CN113164768A | 2021-07-23 | |||
| CN105452463A | 2016-03-30 | |||
| CN107429250A | 2017-12-01 | |||
| CN110573619A | 2019-12-13 | |||
| US20140194489A1 | 2014-07-10 |
Attorney, Agent or Firm:
GUANGZHOU PRIME IP LAW OFFICE (CN)
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