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1.发明授权Compositions and method for inhibiting hepcidin antimicrobial peptide (HAMP) or HAMP-related gene expression 有权抑制铁调素抗菌肽（HAMP）或HAMP相关基因表达的组合物和方法公开(公告)号：US09228188B2
公开(公告)日：2016-01-05
申请号：US14118489
申请日：2012-06-21
申请人： Brian Bettencourt , Akin Akinc , Alfica Sehgal , Don Foster , Stuart Milstein , Satyanarayana Kuchimanchi , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev
发明人： Brian Bettencourt , Akin Akinc , Alfica Sehgal , Don Foster , Stuart Milstein , Satyanarayana Kuchimanchi , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev
IPC分类号： C12N15/11 , C12N15/113 , A61K31/7105 , A61K33/26 , A61K47/48
CPC分类号： C12N15/113 , A61K31/7105 , A61K33/26 , A61K47/48092 , A61K47/549 , C12N15/1136 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3233 , C12N2310/351 , C12N2310/3515 , C12N2310/3521 , C12N2310/3533 , C12N2320/30
摘要： The invention relates to lipid formulated double-stranded ribonucleic acid (dsRNA) targeting a hepcidin antimicrobial peptide (HAMP) and/or HAMP-related gene, and methods of using the dsRNA to inhibit expression of HAMP and/or HAMP-related genes.
摘要翻译： 本发明涉及靶向铁调素抗体肽（HAMP）和/或HAMP相关基因的脂质配制双链核糖核酸（dsRNA），以及使用dsRNA抑制HAMP和/或HAMP相关基因表达的方法。
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2.发明申请Compositions and Method for Inhibiting Hepcidin Antimicrobial Peptide (HAMP) or HAMP-Related Gene Expression 有权抑制Hepcidin抗菌肽（HAMP）或HAMP相关基因表达的组合物和方法公开(公告)号：US20140127325A1
公开(公告)日：2014-05-08
申请号：US14118489
申请日：2012-06-21
申请人： Brian Bettencourt , Akin Akinc , Alfica Sehgal , Don Foster , Stuart Milstein , Satyanarayana Kuchimanchi , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev
发明人： Brian Bettencourt , Akin Akinc , Alfica Sehgal , Don Foster , Stuart Milstein , Satyanarayana Kuchimanchi , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev
IPC分类号： C12N15/113 , A61K47/48 , A61K33/26 , A61K31/7105
CPC分类号： C12N15/113 , A61K31/7105 , A61K33/26 , A61K47/48092 , A61K47/549 , C12N15/1136 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3233 , C12N2310/351 , C12N2310/3515 , C12N2310/3521 , C12N2310/3533 , C12N2320/30
摘要： The invention relates to lipid formulated double-stranded ribonucleic acid (dsRNA) targeting a hepcidin antimicrobial peptide (HAMP) and/or HAMP-related gene, and methods of using the dsRNA to inhibit expression of HAMP and/or HAMP-related genes.
摘要翻译： 本发明涉及靶向铁调素抗体肽（HAMP）和/或HAMP相关基因的脂质配制双链核糖核酸（dsRNA），以及使用dsRNA抑制HAMP和/或HAMP相关基因表达的方法。
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3.发明申请Compositions and Methods for Inhibition of Expression of Protein C (PROC) Genes 有权抑制蛋白C（PROC）基因表达的组成和方法公开(公告)号：US20140142162A1
公开(公告)日：2014-05-22
申请号：US14127090
申请日：2012-06-21
申请人： Ivanka Toudjarska , John M. Maraganore , Brian Bettencourt , Stuart Milstein , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev , Satyanarayana Kuchimanchi
发明人： Ivanka Toudjarska , John M. Maraganore , Brian Bettencourt , Stuart Milstein , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev , Satyanarayana Kuchimanchi
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) targeting a PROC gene, and methods of using the dsRNA to inhibit expression of PROC. At least one nucleotide of the dsRNA can be a modified nucleotide, e.g., a 2-0-methyl modified nucleotide, a nucleotide comprising a 5′-phosphorothioate group, and a terminal nucleotide linked to a cholesteryl derivative or dodecanoic acid bisdecylamide group. Other examples of modified nucleotides include a 2′-deoxy-2′-fluoro modified nucleotide, a 2′-deoxymodified nucleotide, a locked nucleotide, an abasic nucleotide, 2′-amino-modified nucleotide, 2′-alkyl-modified nucleotide, morpholino nucleotide, a phosphoramidate, and a non-natural base comprising nucleotide. A dsRNA of the invention can include one or more of any of these modified nucleotides.
摘要翻译： 本发明涉及靶向PROC基因的双链核糖核酸（dsRNA）以及使用dsRNA抑制PROC表达的方法。 dsRNA的至少一个核苷酸可以是修饰的核苷酸，例如2-0-甲基修饰的核苷酸，包含5'-硫代磷酸酯基团的核苷酸，以及与胆固醇衍生物或十二烷酸双癸酰胺基团连接的末端核苷酸。 修饰的核苷酸的其它实例包括2'-脱氧-2'-氟修饰的核苷酸，2'-脱氧改性的核苷酸，锁定的核苷酸，脱碱核苷酸，2'-氨基修饰的核苷酸，2'-烷基修饰的核苷酸， 吗啉代核苷酸，氨基磷酸酯和包含核苷酸的非天然碱基。 本发明的dsRNA可以包括这些修饰的核苷酸中的一种或多种。
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4.发明授权Compositions and methods for inhibition of expression of protein C (PROC) genes 有权抑制蛋白C（PROC）基因表达的组合物和方法公开(公告)号：US09068184B2
公开(公告)日：2015-06-30
申请号：US14127090
申请日：2012-06-21
申请人： Ivanka Toudjarska , John M. Maraganore , Brian Bettencourt , Stuart Milstein , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev , Satyanarayana Kuchimanchi
发明人： Ivanka Toudjarska , John M. Maraganore , Brian Bettencourt , Stuart Milstein , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev , Satyanarayana Kuchimanchi
IPC分类号： A61K48/00 , C12N15/11 , C07H21/02 , C07H21/04 , C12N15/113
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) targeting a PROC gene, and methods of using the dsRNA to inhibit expression of PROC. At least one nucleotide of the dsRNA can be a modified nucleotide, e.g., a 2-0-methyl modified nucleotide, a nucleotide comprising a 5′-phosphorothioate group, and a terminal nucleotide linked to a cholesteryl derivative or dodecanoic acid bisdecylamide group. Other examples of modified nucleotides include a 2′-deoxy-2′-fluoro modified nucleotide, a 2′-deoxymodified nucleotide, a locked nucleotide, an abasic nucleotide, 2′-amino-modified nucleotide, 2′-alkyl-modified nucleotide, morpholino nucleotide, a phosphoramidate, and a non-natural base comprising nucleotide. A dsRNA of the invention can include one or more of any of these modified nucleotides.
摘要翻译： 本发明涉及靶向PROC基因的双链核糖核酸（dsRNA）以及使用dsRNA抑制PROC表达的方法。 dsRNA的至少一个核苷酸可以是修饰的核苷酸，例如2-0-甲基修饰的核苷酸，包含5'-硫代磷酸酯基团的核苷酸，以及与胆固醇衍生物或十二烷酸双癸酰胺基团连接的末端核苷酸。 修饰的核苷酸的其它实例包括2'-脱氧-2'-氟修饰的核苷酸，2'-脱氧改性的核苷酸，锁定的核苷酸，脱碱核苷酸，2'-氨基修饰的核苷酸，2'-烷基修饰的核苷酸， 吗啉代核苷酸，氨基磷酸酯和包含核苷酸的非天然碱基。 本发明的dsRNA可以包括这些修饰的核苷酸中的一种或多种。
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公开(公告)号：US09051567B2
公开(公告)日：2015-06-09
申请号：US13378346
申请日：2010-06-15
IPC分类号： A61K31/713 , A61K9/127 , A61P3/06 , C12N15/11 , C12N15/113 , C12N9/64
CPC分类号： C12N15/113 , A61K9/127 , A61K31/366 , A61K31/567 , A61K31/7088 , C12N9/6454 , C12N15/111 , C12N15/1135 , C12N15/1136 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2320/31 , C12N2320/32 , C12Y304/21061
摘要： This invention relates to methods for increasing the efficiency of siRNA administrations via pre-administration of an agent that increases LDL receptor levels.
摘要翻译： 本发明涉及通过预先施用增加LDL受体水平的试剂来提高siRNA施用效率的方法。
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公开(公告)号：US20120244207A1
公开(公告)日：2012-09-27
申请号：US13378346
申请日：2010-06-15
IPC分类号： A61K31/713 , A61P3/06 , A61K9/127
CPC分类号： C12N15/113 , A61K9/127 , A61K31/366 , A61K31/567 , A61K31/7088 , C12N9/6454 , C12N15/111 , C12N15/1135 , C12N15/1136 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2320/31 , C12N2320/32 , C12Y304/21061
摘要： This invention relates to methods for increasing the efficiency of siRNA administrations via pre-administration of an agent that lowers LDL receptor levels.
摘要翻译： 本发明涉及通过预先施用降低LDL受体水平的试剂来提高siRNA施用效率的方法。
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7.发明授权Oligomers comprising activated disulfides which bind to plasma proteins and their use for delivery to cells 有权包含结合血浆蛋白的活性二硫化物的低聚物及其用于递送至细胞的用途公开(公告)号：US07713944B2
公开(公告)日：2010-05-11
申请号：US11251564
申请日：2005-10-13
CPC分类号： A61K47/62 , A61K47/549
摘要： The activated oligomer compounds described herein are capable of forming bio-reversible covalent bonds with plasma proteins, in particular with human serum albumin. The plasma protein-oligomer complexes of the present invention exhibit enhanced cellular entry and significantly enhanced serum half-life.
摘要翻译： 本文所述的活化的低聚物化合物能够与血浆蛋白质，特别是与人血清白蛋白形成生物可逆共价键。 本发明的血浆蛋白质 - 低聚物复合物表现出增强的细胞进入和显着增强的血清半衰期。
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8.发明授权Compounds, processes and intermediates for synthesis of mixed backbone oligomeric compounds 有权用于合成混合骨架低聚化合物的化合物，方法和中间体公开(公告)号：US06462184B2
公开(公告)日：2002-10-08
申请号：US09726096
申请日：2000-11-29
申请人： Muthiah Manoharan , Martin A. Maier
发明人： Muthiah Manoharan , Martin A. Maier
IPC分类号： C07H1900
CPC分类号： C07H21/00 , C07B2200/11
摘要： Synthetic processes are provided wherein mixed backbone oligomeric compounds are prepared having at least one phosphodiester internucleoside linkage in addition to one or more phosphorothioate, phosphoramidate and boranophosphate internucleoside linkages. Novel H-phosphonate intermediates are also disclosed that are useful in the synthetic processes. The synthetic processes use a novel oxidation step to oxidize H-phosphonate internucleoside linkages to phosphodiester internucleoside linkages without degradation of adjacent phosphorothioate, phosphoramidate and boranophosphate internucleoside linkages. Also provided are synthetic intermediates useful in such processes.
摘要翻译： 提供合成方法，其中制备除了一种或多种硫代磷酸酯，氨基磷酸酯和硼磷酸酯核苷间键外还具有至少一个磷酸二酯核苷间键的混合骨架低聚化合物。 还公开了可用于合成方法的新型H-膦酸酯中间体。 合成方法使用新的氧化步骤将H-膦酸酯核苷间键合氧化成磷酸二酯核苷间键而不降解相邻的硫代磷酸酯，氨基磷酸酯和硼酸三磷酸核苷间键。 还提供了可用于这些方法的合成中间体。
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公开(公告)号：US06878805B2
公开(公告)日：2005-04-12
申请号：US10222595
申请日：2002-08-16
申请人： Muthiah Manoharan , Martin A. Maier
发明人： Muthiah Manoharan , Martin A. Maier
CPC分类号： C07K9/00 , C07K14/003 , C12N15/113 , C12N2310/3181 , C12N2310/3513 , C12N2310/3515
摘要： The present invention relates to novel amphipathic peptide-conjugated oligomeric compounds and to methods of making and using such compounds. The invention further relates to methods of enhancing the cellular uptake of oligomeric compounds comprising conjugating the compounds to amphipathic moieties such as amphipathic peptides.
摘要翻译： 本发明涉及新型两亲肽 - 共轭寡聚化合物以及制备和使用这些化合物的方法。 本发明还涉及增强寡聚化合物的细胞摄取的方法，包括将化合物缀合到两亲性部分如两亲肽。
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10.发明授权Compounds, processes and intermediates for synthesis of mixed backbone oligomeric compounds 失效用于合成混合骨架低聚化合物的化合物，方法和中间体公开(公告)号：US06207819B1
公开(公告)日：2001-03-27
申请号：US09250075
申请日：1999-02-12
申请人： Muthiah Manoharan , Martin A. Maier
发明人： Muthiah Manoharan , Martin A. Maier
IPC分类号： C07H2100
CPC分类号： C07H21/00 , C07B2200/11
摘要： Synthetic processes are provided wherein mixed backbone oligomeric compounds are prepared having at least one phosphodiester intemucleoside linkage in addition to one or more phosphorothioate, phosphoramidate and boranophosphate internucleoside linkages. Novel H-phosphonate intermediates are also disclosed that are useful in the synthetic processes. The synthetic processes use a novel oxidation step to oxidize H-phosphonate internuleoside linkages to phosphodiester internuleoside linkages without degradation of adjacent phosphorothioate, phosphoramidate and boranophosphate internucleoside linkages. Also provided are synthetic intermediates useful in such processes.
摘要翻译： 提供了合成方法，其中除了一种或多种硫代磷酸酯，氨基磷酸酯和硼酸三磷酸核苷间键外，制备具有至少一个磷酸二酯核苷键的混合骨架低聚化合物。 还公开了可用于合成方法的新型H-膦酸酯中间体。 合成方法使用新的氧化步骤将H-磷酸酯间隔蛋白键连接到磷酸二酯间隔键而不降解相邻的硫代磷酸酯，氨基磷酸酯和硼酸三磷酸核苷间键。 还提供了可用于这些方法的合成中间体。
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