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公开(公告)号：US11408001B1
公开(公告)日：2022-08-09
申请号：US17347863
申请日：2021-06-15
发明人： Kevin Fitzgerald , William Querbes , James Butler , Stephanie Williams , Abigail Liebow , Gregory Hinkle , Martin A. Maier , Stuart Milstein , Satyanarayana Kuchimanchi , Muthiah Manoharan
IPC分类号： C12N15/113 , A61K31/713 , A61P3/04 , A61P43/00 , A61P13/12 , A61P1/18 , A61P3/10 , A61P9/12 , A61P15/08 , A61P9/10 , A61P1/16 , A61P3/06
摘要： The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the apolipoprotein C3 (APOC3) gene, and methods of using such RNAi agents to inhibit expression of APOC3 and methods of treating subjects having an APOC3 associated disorder, e.g., hypertriglyceridemia.
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2.发明申请公开(公告)号：US20170304446A1
公开(公告)日：2017-10-26
申请号：US15517471
申请日：2015-10-09
IPC分类号： A61K45/06 , A61K31/713 , A61K31/00 , A61K45/00
CPC分类号： A61K45/06 , A61K31/00 , A61K31/713 , A61K45/00 , A61K47/549 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/344 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12Y101/03015 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： The invention relates to RNAi agents, e.g. double-stranded RNAi agents, targeting the HAO1 gene, and method of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1. Described herein are double-stranded RNAi agents which inhibit the expression of a HA01 gene in a cell, such as a cell within a subject, e.g., a mammal, such as a human having a HAO1 associated disorder, and uses of such double-stranded RNAi agents. In certain aspects of the invention, substantially all of the nucleotides of an iRNA of the invention are modified.
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公开(公告)号：US20170260527A1
公开(公告)日：2017-09-14
申请号：US15597225
申请日：2017-05-17
发明人： Kevin Fitzgerald , William Querbes , James Butler , Stephanie Williams , Abigail Liebow , Gregory Hinkle , Martin Maier , Stuart Milstein , Satyanarayana Kuchimanchi , Muthiah Manoharan
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/335 , C12N2310/343 , C12N2310/3515 , C12N2310/3521 , C12N2310/3533
摘要： The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the apolipoprotein C3 (APOC3) gene, and methods of using such RNAi agents to inhibit expression of APOC3 and methods of treating subjects having an APOC3 associated disorder, e.g., hypertriglyceridemia.
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公开(公告)号：US20230121944A1
公开(公告)日：2023-04-20
申请号：US17819691
申请日：2022-08-15
IPC分类号： A61K45/06 , A61K31/713 , C12N15/113 , A61K47/54 , A61K31/00 , A61K45/00
摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the HAO1 gene, and methods of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1.
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公开(公告)号：US11446380B2
公开(公告)日：2022-09-20
申请号：US16673863
申请日：2019-11-04
IPC分类号： C07H21/02 , C07H21/04 , A61K45/06 , A61K47/54 , C12N15/113 , A61K31/713 , A61K31/00 , A61K45/00
摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the HAO1 gene, and methods of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1.
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6.发明申请公开(公告)号：US20200316201A1
公开(公告)日：2020-10-08
申请号：US16673863
申请日：2019-11-04
IPC分类号： A61K45/06 , A61K47/54 , C12N15/113 , A61K31/713
摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the HAO1 gene, and methods of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1.
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公开(公告)号：US20210228614A1
公开(公告)日：2021-07-29
申请号：US17106259
申请日：2020-11-30
发明人： David Erbe , Abigail Liebow , Kevin Fitzgerald , Gregory Hinkle , Kyle David Wood , Ross Philip Holmes , John Knight
IPC分类号： A61K31/7105 , C12N15/113
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LDHA gene, as well as methods of inhibiting expression of LDHA, methods of inhibiting LDHA and HAO1, and methods of treating subjects that would benefit from reduction in expression of LDHA, such as subjects having an oxalate pathway-associated disease, disorder, or condition, using such dsRNA compositions.
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公开(公告)号：US11034958B2
公开(公告)日：2021-06-15
申请号：US17128320
申请日：2020-12-21
发明人： Kevin Fitzgerald , William Querbes , James Butler , Stephanie Williams , Abigail Liebow , Gregory Hinkle , Martin A. Maier , Stuart Milstein , Satyanarayana Kuchimanchi , Muthiah Manoharan
IPC分类号： C12N15/113
摘要： The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the apolipoprotein C3 (APOC3) gene, and methods of using such RNAi agents to inhibit expression of APOC3 and methods of treating subjects having an APOC3 associated disorder, e.g., hypertriglyceridemia.
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公开(公告)号：US20200206258A1
公开(公告)日：2020-07-02
申请号：US16811476
申请日：2020-03-06
发明人： David Erbe , Abigail Liebow , Kevin Fitzgerald , Gregory Hinkle , Kyle David Wood , Ross Philip Holmes , John Knight
IPC分类号： A61K31/7105 , C12N15/113
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LDHA gene, as well as methods of inhibiting expression of LDHA, methods of inhibiting LDHA and HAO1, and methods of treating subjects that would benefit from reduction in expression of LDHA, such as subjects having an oxalate pathway-associated disease, disorder, or condition, using such dsRNA compositions.
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公开(公告)号：US10407679B2
公开(公告)日：2019-09-10
申请号：US15597225
申请日：2017-05-17
发明人： Kevin Fitzgerald , William Querbes , James Butler , Stephanie Williams , Abigail Liebow , Gregory Hinkle , Martin Maier , Stuart Milstein , Satyanarayana Kuchimanchi , Muthiah Manoharan
IPC分类号： C12N15/113
摘要： The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the apolipoprotein C3 (APOC3) gene, and methods of using such RNAi agents to inhibit expression of APOC3 and methods of treating subjects having an APOC3 associated disorder, e.g., hypertriglyceridemia.
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