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公开(公告)号：US20230002772A1
公开(公告)日：2023-01-05
申请号：US17586850
申请日：2022-01-28
发明人： Anna Borodovsky , Kallanthottathil G. Rajeev , Kevin Fitzgerald , Maria Frank-Kamenetsky , William Querbes , Martin A. Maier , Klaus Charisse , Satyanarayana Kuchimanchi , Muthiah Manoharan , Stuart Milstein
IPC分类号： C12N15/113 , A61K31/713 , A61K47/54
摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the PCSK9 gene, and methods of using such RNAi agents to inhibit expression of PCSK9 and methods of treating subjects having a lipid disorder, such as a hyperlipidemia.
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公开(公告)号：US11406716B2
公开(公告)日：2022-08-09
申请号：US16850555
申请日：2020-04-16
发明人： Kallanthottathil G. Rajeev , Tracy Zimmermann , Muthiah Manoharan , Martin Maier , Satyanarayana Kuchimanchi , Klaus Charisse
IPC分类号： C12N15/113 , A61K48/00 , C07H21/02 , C12N15/85
摘要： One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene. The dsRNA duplex comprises one or more motifs of three identical modifications on three consecutive nucleotides in one or both strand, particularly at or near the cleavage site of the strand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
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3.发明申请公开(公告)号：US20180362978A1
公开(公告)日：2018-12-20
申请号：US15948624
申请日：2018-04-09
发明人： Brian Bettencourt , Kevin Fitzgerald , Stuart Milstein , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev , Satyanarayana Kuchimanchi , Muthiah Manoharan , Tuyen Nguyen
IPC分类号： C12N15/113
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) targeting an APOC3 gene, and methods of using the dsRNA to inhibit expression of APOC3.
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公开(公告)号：US10125369B2
公开(公告)日：2018-11-13
申请号：US14650128
申请日：2013-12-05
发明人： Anna Borodovsky , Kallanthottathil G. Rajeev , Kevin Fitzgerald , Maria Frank-Kamenetsky , William Querbes , Martin Maier , Klaus Charisse , Satyanarayana Kuchimanchi , Muthiah Manoharan , Stuart Milstein
IPC分类号： C07H21/04 , A61K48/00 , C12N15/11 , C12N15/113 , A61K31/713 , A61K47/54
摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the PCSK9 gene, and methods of using such RNAi agents to inhibit expression of PCSK9 and methods of treating subjects having a lipid disorder, such as a hyperlipidemia.
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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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6.发明授权公开(公告)号：US09249415B2
公开(公告)日：2016-02-02
申请号：US14699140
申请日：2015-04-29
发明人： Kevin Fitzgerald , James Butler , Brian Bettencourt , Anna Borodovsky , Satyanarayana Kuchimanchi , Klaus Charisse , Muthiah Manoharan , Martin Maier , Kallanthottathil G. Rajeev , Donald Foster
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K31/713 , A61K39/3955 , C07K16/18 , C07K2317/24 , C07K2317/76 , C12N2310/14 , C12N2310/3125 , C12N2310/315 , C12N2310/321 , C12N2310/351 , C12N2310/3527 , C12N2320/30
摘要： The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.
摘要翻译： 本发明涉及iRNA，例如双链核糖核酸（dsRNA），针对补体成分C5基因的组合物，以及使用这种iRNA的方法，例如dsRNA，组合物抑制C5的表达并治疗具有补体成分的受试者 C5相关疾病，例如阵发性夜间血红蛋白尿。
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公开(公告)号：US20160017335A1
公开(公告)日：2016-01-21
申请号：US14650128
申请日：2013-12-05
发明人： Anna Borodovsky , Rajeev G. Kallanthottathil , Kevin Fitzgerald , Maria Frank-Kamenetsky , William Querbes , Martin Maier , Klaus Charisse , Satyanarayana Kuchimanchi , Muthiah Manoharan , Stuart Milstein
IPC分类号： C12N15/113 , A61K31/713 , A61K47/48
CPC分类号： C12N15/1137 , A61K31/713 , A61K47/549 , C12N2310/14 , C12N2310/312 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/343 , C12N2310/351 , C12N2310/3515 , C12N2320/35 , C12Y304/21 , C12N2310/3533
摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the PCSK9 gene, and methods of using such RNAi agents to inhibit expression of PCSK9 and methods of treating subjects having a lipid disorder, such as a hyperlipidemia.
摘要翻译： 本发明涉及靶向PCSK9基因的RNAi试剂，例如双链RNAi试剂，以及使用这种RNAi试剂抑制PCSK9表达的方法和治疗患有脂质疾病如高脂血症的受试者的方法。
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公开(公告)号：US20160017330A1
公开(公告)日：2016-01-21
申请号：US14806084
申请日：2015-07-22
发明人： Akin Akinc , Alfica Sehgal , Ivanka Toudjarska , Donald Foster , Stuart Milstein , Brian Bettencourt , Martin Maier , Klaus Charisse , Satyanarayana Kuchimanchi , Kallanthottathil G. Rajeev , Muthiah Manoharan
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K31/713 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/335 , C12N2310/351 , C12N2310/3515 , C12N2310/3521 , C12N2310/3533 , C12N2320/30 , C12N2310/3525 , C12N2310/3523
摘要： The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and methods of treating subjects having a bleeding disorder, such as a hemophilia.
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公开(公告)号：US11873491B2
公开(公告)日：2024-01-16
申请号：US17372710
申请日：2021-07-12
发明人： Kevin Fitzgerald , James Butler , Brian Bettencourt , Anna Borodovsky , Satyanarayana Kuchimanchi , Klaus Charisse , Muthiah Manoharan , Martin A. Maier , Kallanthottathil G. Rajeev , Donald Foster
IPC分类号： C12N15/113 , A61K31/713 , A61K39/395 , C07K16/18
CPC分类号： C12N15/113 , A61K31/713 , A61K39/3955 , C07K16/18 , C07K2317/24 , C07K2317/76 , C12N2310/14 , C12N2310/315 , C12N2310/3125 , C12N2310/321 , C12N2310/351 , C12N2310/3527 , C12N2320/30
摘要： The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.
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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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