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    Targeting Ligands
    1.
    发明申请
    Targeting Ligands 有权

    公开(公告)号：US20220033817A1

    公开(公告)日：2022-02-03

    申请号：US17351169

    申请日：2021-06-17

    申请人： Arrowhead Pharmaceuticals, Inc.

    发明人： Zhen Li ,  Tao Pei ,  Agnieszka Glebocka ,  Michael Lawler ,  Fred Fleitz ,  Erich Altenhofer ,  Pankaj Kumar

    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.

    RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
    2.
    发明授权
    RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3) 有权

    公开(公告)号：US11214801B2

    公开(公告)日：2022-01-04

    申请号：US16778188

    申请日：2020-01-31

    申请人： Arrowhead Pharmaceuticals, Inc.

    发明人： Zhen Li ,  Rui Zhu ,  Tao Pei ,  Steven Kanner ,  So Wong

    IPC分类号： C12N15/113 ,  C07H3/02 ,  A61P3/04 ,  A61P1/18 ,  A61P9/10 ,  A61K9/00

    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 agents in vivo provides 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.

    RNAi Agents for Inhibiting Expression of Alpha-ENaC And Methods of Use
    4.
    发明申请
    RNAi Agents for Inhibiting Expression of Alpha-ENaC And Methods of Use 审中-公开

    公开(公告)号：US20200299691A1

    公开(公告)日：2020-09-24

    申请号：US16778582

    申请日：2020-01-31

    申请人： Arrowhead Pharmaceuticals, Inc.

    发明人： Zhen Li ,  Rui Zhu ,  Tao Pei ,  Anthony Nicholas ,  Erik W Bush

    IPC分类号： C12N15/113 ,  A61P5/40

    摘要： Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of an alpha-ENaC (SCNN1A) gene. The alpha-ENaC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an alpha-ENaC gene. Pharmaceutical compositions that include one or more alpha-ENaC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described alpha-ENaC RNAi agents to epithelial cells, such as pulmonary epithelial cells, in vivo, provides for inhibition of alpha-ENaC gene expression and a reduction in ENaC activity, which can provide a therapeutic benefit to subjects, including human subjects.

    RNAi Agents And Compositions for Inhibiting Expression of Apolipoprotein C-III (APOC3)
    5.
    发明申请
    RNAi Agents And Compositions for Inhibiting Expression of Apolipoprotein C-III (APOC3) 审中-公开

    公开(公告)号：US20200299690A1

    公开(公告)日：2020-09-24

    申请号：US16778188

    申请日：2020-01-31

    申请人： Arrowhead Pharmaceuticals, Inc.

    发明人： Zhen Li ,  Rui Zhu ,  Tao Pei ,  Steven Kanner ,  So Wong

    IPC分类号： C12N15/113 ,  C07H3/02 ,  A61P3/04 ,  A61P1/18 ,  A61P9/10

    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 agents in vivo provides 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.

    Targeting ligands for therapeutic compounds
    9.
    发明授权
    Targeting ligands for therapeutic compounds 有权

    公开(公告)号：US10246709B2

    公开(公告)日：2019-04-02

    申请号：US15452423

    申请日：2017-03-07

    申请人： Arrowhead Pharmaceuticals, Inc.

    发明人： David B. Rozema ,  Darren H. Wakefield ,  Andrei V. Blokhin ,  Jonathan D. Benson ,  Zhen Li ,  Tao Pei ,  Fred Fleitz

    IPC分类号： C12N15/113 ,  A61K31/713 ,  C07H15/04 ,  A61K47/54

    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 target in vivo. 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 an expression-inhibiting oligomeric 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.

    RNAi Agents for Inhibiting Expression of Alpha-ENaC And Methods of Use
    10.
    发明申请
    RNAi Agents for Inhibiting Expression of Alpha-ENaC And Methods of Use 审中-公开

    公开(公告)号：US20190010494A1

    公开(公告)日：2019-01-10

    申请号：US16028006

    申请日：2018-07-05

    申请人： Arrowhead Pharmaceuticals, Inc.

    发明人： Zhen Li ,  Rui Zhu ,  Tao Pei ,  Anthony Nicholas ,  Erik W Bush

    IPC分类号： C12N15/113 ,  A61P5/40

    摘要： Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of an alpha-ENaC (SCNN1A) gene. The alpha-ENaC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an alpha-ENaC gene. Pharmaceutical compositions that include one or more alpha-ENaC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described alpha-ENaC RNAi agents to epithelial cells, such as pulmonary epithelial cells, in vivo, provides for inhibition of alpha-ENaC gene expression and a reduction in ENaC activity, which can provide a therapeutic benefit to subjects, including human subjects.