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41.发明公开
公开(公告)号：EP3312281A2
公开(公告)日：2018-04-25
申请号：EP17187047.0
申请日：2014-03-13
发明人： FITZGERALD, Kevin , BUTLER, James , BETTENCOURT, Brian , BORODOVSKY, Anna , KUCHIMANCHI, Satyanarayana , CHARISSE, Klaus , MANOHARAN, Muthiah , MAIER, Martin , RAJEEV, Kallanthottahil G.
IPC分类号： C12N15/113 , A61K31/7088
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： 本发明涉及靶向补体成分C5基因的iRNA（例如双链核糖核酸（dsRNA））组合物，以及使用这种iRNA例如dsRNA组合物抑制C5的表达并治疗具有补体成分 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：EP2929031B1
公开(公告)日：2017-10-25
申请号：EP13811327.9
申请日：2013-12-05
发明人： BORODOVSKY, Anna , KALLANTHOTTATHIL, Rajeev G. , FITZGERALD, Kevin , FRANK-KAMENETSKY, Maria , QUERBES, William , MAIER, Martin , CHARISSE, Klaus , KUCHIMANCHI, Satyanarayana , MANOHARAN, Muthiah , MILSTEIN, Stuart
IPC分类号： C12N15/113 , A61K31/713
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：EP2970974B1
公开(公告)日：2017-08-23
申请号：EP14722018.0
申请日：2014-03-13
发明人： FITZGERALD, Kevin , BUTLER, James , BETTENCOURT, Brian , BORODOVSKY, Anna , KUCHIMANCHI, Satyanarayana , CHARISSE, Klaus , MANOHARAN, Muthiah , MAIER, Martin , RAJEEV, Kallanthottathil, G. , FOSTER, Donald
IPC分类号： C12N15/113 , A61K31/7088
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
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公开(公告)号：EP2633046A2
公开(公告)日：2013-09-04
申请号：EP11837272.1
申请日：2011-10-31
CPC分类号： A61K31/713 , C12N15/1137 , C12N2310/111 , C12N2310/14 , C12N2310/314 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3233 , C12N2310/332 , C12N2310/3515 , C12Y304/21061 , C12N2310/3521 , C12N2310/3527 , C12N2310/3533 , C12N2310/3531
摘要： The invention relates to siRNAs targeting a PCSK9 gene, and methods of using siRNAs to inhibit expression of PC-SK9 and to treat PCSK9 related disorders, e.g., hyperlipidemia.
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公开(公告)号：EP2616543A1
公开(公告)日：2013-07-24
申请号：EP11767103.2
申请日：2011-09-14
发明人： RAJEEV, Kallanthottathil, G. , ZIMMERMANN, Tracy , MANOHARAN, Muthiah , MAIER, Martin , FITZGERALD, Kevin
IPC分类号： C12N15/113 , A61K31/713 , C12N15/87 , C07H21/00
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/322 , C12N2310/343 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/32 , C12N2320/52 , C12N2310/3533
摘要： The present invention provides effective motifs for RNA agents conjugated to at least one ligand, which are advantageous for the
in vivo delivery of iRNA duplex agents. Additionally, the present invention provides methods of making these compositions, as well as methods of introducing these iRNA duplex agents into cells using these compositions, e.g., for the treatment of various disease conditions.摘要翻译： 本发明提供了与至少一种配体结合的RNA试剂的有效基序，其有利于体内递送iRNA双链体试剂。 另外，本发明提供了制备这些组合物的方法，以及使用这些组合物将这些iRNA双链体试剂引入细胞中的方法，例如用于治疗各种疾病状态的方法。
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公开(公告)号：EP4083209A1
公开(公告)日：2022-11-02
申请号：EP22162358.0
申请日：2013-12-05
发明人： BORODOVSKY, Anna , KALLANTHOTTATHIL, Rajeev G. , FITZGERALD, Kevin , FRANK-KAMENETSKY, Maria , QUERBES, William , MAIER, Martin , CHARISSE, Klaus , KUCHIMANCHI, Satyanarayana , MANOHARAN, Muthiah , MILSTEIN, Stuart
IPC分类号： C12N15/113 , A61K31/713
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：EP3960860A3
公开(公告)日：2022-06-08
申请号：EP21180120.4
申请日：2015-02-11
IPC分类号： C12N15/113 , A61K31/713
摘要： The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the ketohexokinase (KHK) gene, and methods of using such RNAi agents to inhibit expression of KHK and methods of treating subjects having a KHK-associated disorder, e.g., liver disease (e.g., fatty liver, steatohepatitis), dyslipidemia (e.g., hyperlipidemia, high LDL cholesterol, low HDL cholesterol, hypertriglyceridemia, postprandial hypertriglyceridemia), disorders of glycemic control (e.g., insulin resistance, diabetes), cardiovascular disease (e.g., hypertension, endothelial cell dysfunction), kidney disease (e.g., acute kidney disorder, tubular dysfunction, proinflammatory changes to the proximal tubules), metabolic syndrome, adipocyte dysfunction, visceral adipose deposition, obesity, hyperuricemia, gout, eating disorders, and excessive sugar craving.
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公开(公告)号：EP3898979A2
公开(公告)日：2021-10-27
申请号：EP19900781.6
申请日：2019-12-19
发明人： MILSTEIN, Stuart , BROWN, Kirk , NAIR, Jayaprakash , MAIER, Martin , JADHAV, Vasant , KEATING, Mark , CASTORENO, Adam , HASLETT, Patrick , SOUNDARAPANDIAN, Mangala, Meenakshi , FITZGERALD, Kevin
IPC分类号： C12N15/113 , A61P25/28
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50.发明公开
公开(公告)号：EP3856907A1
公开(公告)日：2021-08-04
申请号：EP19784188.5
申请日：2019-09-26
发明人： NAIR, Jayaprakash, K. , MAIER, Martin, A.. , JADHAV, Vasant, R. , KEATING, Mark , FITZGERALD, Kevin , MILSTEIN, Stuart , BROWN, Kirk , MANOHARAN, Muthiah
IPC分类号： C12N15/113 , A61K31/713
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