公开(公告)号：US11840688B2

公开(公告)日：2023-12-12

申请号：US17179605

申请日：2021-02-19

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： So Wong ,  David L. Lewis ,  David B. Rozema ,  Darren H. Wakefield ,  Steven B. Kanner ,  Weijun Cheng ,  Lauren J. Almeida ,  Andrei V. Blokhin ,  Jeffrey C. Carlson ,  Anthony L. Nicholas ,  Aaron Almeida ,  Jonathan D. Benson ,  Justin Woods

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K45/06 ,  A61K47/60 ,  A61K47/64

CPC分类号： C12N15/113 ,  A61K31/713 ,  A61K45/06 ,  A61K47/60 ,  A61K47/64 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/343 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2310/322 ,  C12N2310/3533 ,  C12N2310/321 ,  C12N2310/3521

摘要： RNA interference (RNAi) triggers and RNAi trigger conjugates for inhibiting the expression of Hif2α (EPAS1) gene are described. Pharmaceutical compositions comprising one or more Hif2α RNAi triggers optionally with one or more additional therapeutics are also described. Delivery of the described Hif2α RNAi triggers to tumor cells in vivo provides for inhibition of Hif2α gene expression and treatment of cancer.

公开(公告)号：US10662427B2

公开(公告)日：2020-05-26

申请号：US15901810

申请日：2018-02-21

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： Stacey Melquist ,  Steven Kanner ,  David B. Rozema ,  David L. Lewis ,  Lauren J. Almeida ,  Darren H. Wakefield ,  Vladimir S. Trubetskoy ,  Tao Pei ,  Zhen Li ,  Aaron Almeida

IPC分类号： C12N15/113

摘要： RNA interference (RNAi) agents and RNAi agent conjugates for inhibiting the expression of the LPA (apo(a)) gene are described. Pharmaceutical compositions comprising one or more LPA RNAi agents optionally with one or more additional therapeutics are also described. Delivery of the described LPA RNAi agents to liver cells in vivo provides for inhibition of LPA gene expression and treatment of cardiovascular and cardiovascular-related diseases.

公开(公告)号：US20190292547A1

公开(公告)日：2019-09-26

申请号：US15766036

申请日：2017-08-04

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： Zhen Li ,  Rui Zhu ,  Christine Wooddell ,  Bruce D. Given ,  Tao Pei ,  David L. Lewis ,  Lauren J. Almeida ,  David B. Rozema ,  Darren H. Wakefield

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61P31/20

摘要： Described are compositions and methods for inhibition of Hepatitis B virus gene expression. RNA interference (RNAi) agents for inhibiting the expression of Hepatitis B virus gene are described. The HBV RNAi agents disclosed herein may be targeted to cells, such as hepatocytes, for example, by using conjugated targeting ligands. Pharmaceutical compositions comprising one or more HBV RNAi agents optionally with one or more additional therapeutics are also described. Delivery of the described HBV RNAi agents to infected liver in vivo provides for inhibition of HBV gene expression and treatment of diseases and conditions associated with HBV infection.

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.

公开(公告)号：US20180023084A1

公开(公告)日：2018-01-25

申请号：US15716809

申请日：2017-09-27

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： Aaron Almeida ,  Andrei V. Blokhin ,  Darren H. Wakefield ,  Jonathan D. Benson ,  David B. Rozema

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2310/3521 ,  C12N2310/3533 ,  C12N2320/50

摘要： Described are improved disulfide-containing alkyne linking agents having have branched disulfides. The improved linking agents exhibit improved stability. The linking agents are useful for attachment of oligonucleotides to targeting groups or delivery agents.

公开(公告)号：US11534453B2

公开(公告)日：2022-12-27

申请号：US17016021

申请日：2020-09-09

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： Christine I. Wooddell ,  David B. Rozema ,  David L. Lewis ,  Darren H. Wakefield ,  Lauren J. Almeida

IPC分类号： A61K31/713 ,  A61K38/16 ,  A61K47/54 ,  A61K47/59

摘要： Described are compositions and methods for inhibition of Hepatitis B virus gene expression. RNA interference (RNAi) triggers and RNAi trigger conjugates for inhibiting the expression of Hepatitis B virus gene are described. Pharmaceutical compositions comprising one or more HBV RNAi triggers optionally with one or more additional therapeutics are also described. Delivery of the described HBV RNAi triggers to infected liver in vivo provides for inhibition of HBV gene expression and treatment.

公开(公告)号：US10927373B2

公开(公告)日：2021-02-23

申请号：US15954316

申请日：2018-04-16

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： So Wong ,  David L. Lewis ,  David B. Rozema ,  Darren H. Wakefield ,  Steven B. Kanner ,  Weijun Cheng ,  Lauren J. Almeida ,  Andrei V. Blokhin ,  Jeffrey C. Carlson ,  Anthony L. Nicholas ,  Aaron Almeida ,  Jonathan D. Benson ,  Justin Woods

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K45/06 ,  A61K47/60 ,  A61K47/64

摘要： RNA interference (RNAi) triggers and RNAi trigger conjugates for inhibiting the expression of Hif2α (EPAS1) gene are described. Pharmaceutical compositions comprising one or more Hif2α RNAi triggers optionally with one or more additional therapeutics are also described. Delivery of the described Hif2α RNAi triggers to tumor cells in vivo provides for inhibition of Hif2α gene expression and treatment of cancer.

公开(公告)号：US10806750B2

公开(公告)日：2020-10-20

申请号：US16148249

申请日：2018-10-01

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： Christine I. Wooddell ,  David B. Rozema ,  David L. Lewis ,  Darren H. Wakefield ,  Lauren J. Almeida

IPC分类号： A61K31/713 ,  A61K38/16 ,  A61K47/54 ,  A61K47/59

摘要： Described are compositions and methods for inhibition of Hepatitis B virus gene expression. RNA interference (RNAi) triggers and RNAi trigger conjugates for inhibiting the expression of Hepatitis B virus gene are described. Pharmaceutical compositions comprising one or more HBV RNAi triggers optionally with one or more additional therapeutics are also described. Delivery of the described HBV RNAi triggers to infected liver in vivo provides for inhibition of HBV gene expression and treatment.

公开(公告)号：US20190211333A1

公开(公告)日：2019-07-11

申请号：US16363108

申请日：2019-03-25

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 ,  C07H15/04 ,  A61K31/713 ,  A61K47/54

CPC分类号： C12N15/113 ,  A61K31/713 ,  A61K47/549 ,  C07H15/04 ,  C12N2310/14 ,  C12N2310/317 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2310/3521 ,  C12N2310/3533 ,  C12N2310/3525

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.

公开(公告)号：US10130651B2

公开(公告)日：2018-11-20

申请号：US15229314

申请日：2016-08-05

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： Christine I. Wooddell ,  David B. Rozema ,  David L. Lewis ,  Darren H. Wakefield ,  Lauren J. Almeida

IPC分类号： A61K31/713 ,  A61K38/16 ,  A61K47/54 ,  A61K47/59

摘要： Described are compositions and methods for inhibition of Hepatitis B virus gene expression. RNA interference (RNAi) triggers and RNAi trigger conjugates for inhibiting the expression of Hepatitis B virus gene are described. Pharmaceutical compositions comprising one or more HBV RNAi triggers optionally with one or more additional therapeutics are also described. Delivery of the described HBV RNAi triggers to infected liver in vivo provides for inhibition of HBV gene expression and treatment.