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RNAi Agents And Compositions for Inhibiting Expression of Asiaglycoprotein Receptor 1
专利权人:
Arrowhead Pharmaceuticals; Inc.
发明人:
Zhen Li,Tao Pei,Rui Zhu,Bruce D. Given,Stacey Melquist
申请号:
US16756440
公开号:
US20200283777A1
申请日:
2018.10.16
申请国别(地区):
US
年份:
2020
代理人:
摘要:
Described herein are compositions and methods for inhibition of Asialoglycoprotein receptor 1 (ASGR1) gene expression. RNA interference (RNAi) agents, e.g., double stranded RNAi agents, and RNAi agent-targeting ligand conjugates for inhibiting the expression of anASGR1 gene are described. Pharmaceutical compositions comprising one or more ASGR1 RNAi agents, optionally with one or more additional therapeutics, are also described. The ASGR1 RNAi agents can be used in methods of treatment of various diseases and conditions, such as cardiometabolic diseases related to elevated non-HDL cholesterol (non-HDL-C) levels, elevated LDL cholesterol (LDL-C) levels, elevated total cholesterol levels, and/or elevated triglyceride (TG) levels.
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