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用於抑制脂蛋白元C-III (APOC3)表現之RNAi試劑及組合物
专利权人:
ARROWHEAD PHARMACEUTICALS; INC.
发明人:
LI, ZHEN,李 䂦,李 䂦,ZHU, RUI,朱锐,朱銳,PEI, TAO,裴 涛,裴 濤,KANNER, STEVEN,坎纳 史蒂芬,坎納 史蒂芬,WONG, SO,黄 素珍,黃 素珍
申请号:
TW107131910
公开号:
TW201920227A
申请日:
2018.09.11
申请国别(地区):
TW
年份:
2019
代理人:
摘要:
The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents, capable of inhibiting Apolipoprotein C-III (also called APOC3, apoC-III, APOC-III, and APO C-III) gene expression, and compositions that include APOC3 RNAi agents. The APOC3 RNAi agents disclosed herein may be conjugated to targeting ligands, including ligands that include N-acetyl-galactosamine, to facilitate the delivery to cells, including to hepatocytes. Pharmaceutical compositions that include one or more APOC3 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the APOC3 RNAi agentsin vivoprovides for inhibition of APOC3 gene expression, and can result in lower triglycerides and/or cholesterol levels in the subject. The APOC3 RNAi agents can be used in methods of treatment of APOC3-related diseases and disorders, including hypertriglyceridemia, cardiovascular disease, and other metabolic-related disorders and diseases.本發明係關於能夠抑制脂蛋白元C-III (亦稱為APOC3、apoC-III、APOC-III及APO C-III)基因表現之RNAi試劑(例如雙股RNAi試劑)以及包括APOC3 RNAi試劑之組合物。本文中所揭示之該等APOC3 RNAi試劑可與靶向配體(包括包含N-乙醯基-半乳糖胺之配體)結合,以促進遞送至細胞(包括肝細胞)中。亦描述包括一或多種APOC3 RNAi試劑之醫藥組合物,其視情況具有一或多種其他治療劑。活體內遞送該等APOC3 RNAi試劑提供對APOC3基因表現之抑制,且可引起個體中之三酸甘油酯及/或膽固醇含量下降。該等APOC3 RNAi試劑可用於APOC3相關疾病及病症之治療方法中,該等疾病及病症包括高三酸甘油酯血症、心血管疾病及其他代謝相關病症及疾病。
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
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