- 专利标题: Targeting Ligands
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申请号: US17351169申请日: 2021-06-17
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公开(公告)号: US20220033817A1公开(公告)日: 2022-02-03
- 发明人: Zhen Li , Tao Pei , Agnieszka Glebocka , Michael Lawler , Fred Fleitz , Erich Altenhofer , Pankaj Kumar
- 申请人: Arrowhead Pharmaceuticals, Inc.
- 申请人地址: US WI Madison
- 专利权人: Arrowhead Pharmaceuticals, Inc.
- 当前专利权人: Arrowhead Pharmaceuticals, Inc.
- 当前专利权人地址: US WI Madison
- 主分类号: C12N15/113
- IPC分类号: C12N15/113 ; A61K31/711 ; C07H21/02 ; A61K31/7008 ; A61K31/7105 ; C07H5/06 ; C07H21/04 ; A61K47/54 ; A61K31/713
摘要:
Described are novel targeting ligands that may be linked to compounds, such therapeutic compounds that are useful in directing the compounds to the in vivo target. The targeting ligands disclosed herein can serve to target expression-inhibiting oligomeric compounds, such as RNAi agents, to liver cells to modulate gene expression. The targeting ligands disclosed herein, when conjugated to a therapeutic compound, may be used in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Compositions including the targeting ligands disclosed herein when linked to expression-inhibiting oligomeric compounds are capable of mediating expression of target nucleic acid sequences in liver cells, such as hepatocytes, which may be useful in the treatment of diseases or conditions that respond to inhibition of gene expression or activity in a cell, tissue, or organism.
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