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Title:
DOUBLE-STRANDED RIBONUCLEIC ACID FOR INHIBITING ANGPTL3 GENE EXPRESSION, AND MODIFIER, CONJUGATE, AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2024/027518
Kind Code:
A1
Abstract:
The present disclosure relates to a double-stranded ribonucleic acid for inhibiting ANGPTL3 gene expression, and a modifier, a conjugate, and use thereof. Specifically, the present disclosure relates to a double-stranded ribonucleic acid, a double-stranded ribonucleic acid modifier, a double-stranded ribonucleic acid conjugate, a pharmaceutical composition and use for inhibiting ANGPTL3 gene expression, and a method for inhibiting intracellular ANGPTL3 gene expression. The double-stranded ribonucleic acid provided in the present disclosure can bind in cells to form an RNA-induced silencing complex (RISC), which cleaves the mRNA transcribed by the ANGPTL3 gene, efficiently and specifically inhibiting the expression of the ANGPTL3 gene. The double-stranded ribonucleic acid is used for treating diseases associated with ANGPTL3 gene-mediated metabolic disorders, and has important application prospects in clinical disease treatment.

Inventors:
WANG SHUCHENG (CN)
HUANG HE (CN)
WANG YAN (CN)
LIN GUOLIANG (CN)
CHAN YUNXIA (CN)
GENG YUXIAN (CN)
WANG XIAOJUN (CN)
RONG MEI (CN)
Application Number:
PCT/CN2023/108634
Publication Date:
February 08, 2024
Filing Date:
July 21, 2023
Export Citation:
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Assignee:
BEIJING WINSUNNY PHARMACEUTICAL CO LTD (CN)
International Classes:
A61K31/713; A61K31/7125; A61P3/00; C07H21/02
Domestic Patent References:
WO2022079222A12022-04-21
WO2022068923A12022-04-07
Foreign References:
CN107743522A2018-02-27
CN111343994A2020-06-26
CN110945130A2020-03-31
CN111973617A2020-11-24
Attorney, Agent or Firm:
LINDA LIU & PARTNERS (CN)
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