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    NOVEL FORMATS FOR ORGANIC COMPOUNDS FOR USE IN RNA INTERFERENCE
    4.
    发明申请
    NOVEL FORMATS FOR ORGANIC COMPOUNDS FOR USE IN RNA INTERFERENCE 审中-公开
    用于RNA干扰的有机化合物的新格式

    公开(公告)号：WO2015051366A3

    公开(公告)日：2015-07-02

    申请号：PCT/US2014059301

    申请日：2014-10-06

    申请人： NOVARTIS AG ,  BARYZA JEREMY LEE ,  BLOMMERS MARCEL ,  CHUTKOW WILLIAM ,  FERNANDEZ CESAR ,  GENO ERIN ,  GOSSERT ALVAR ,  GREENIDGE PAULETTE ,  HUESKEN DIETER ,  HUNZIKER JUERG ,  NATT FRANCOIS JEAN-CHARLES ,  PATNAIK ANUP ,  PATTERSON ANDREW ,  RONDEAU JEAN-MICHEL RENE ,  WEILER JAN ,  ZHU MEICHENG

    发明人： BARYZA JEREMY LEE ,  BLOMMERS MARCEL ,  CHUTKOW WILLIAM ,  FERNANDEZ CESAR ,  GENO ERIN ,  GOSSERT ALVAR ,  GREENIDGE PAULETTE ,  HUESKEN DIETER ,  HUNZIKER JUERG ,  NATT FRANCOIS JEAN-CHARLES ,  PATNAIK ANUP ,  PATTERSON ANDREW ,  RONDEAU JEAN-MICHEL RENE ,  WEILER JAN ,  ZHU MEICHENG

    IPC分类号： C12N15/113

    CPC分类号： C12N15/113 ,  C12N2310/14 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/332 ,  C12N2310/341 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/3533

    摘要： The disclosure relates to compositions comprising a RNAi agent having a novel format including a spacer subunit. The disclosure relates to compositions comprising a RNAi agent having a novel format: an 18-mer format with at least one internal spacer. These RNAi agents comprise a first and a second 18-mer strand, wherein the first strand is 18 ribonucleotides or 18 total ribonucleotides and spacer subunit(s), and the second strand is 18 total ribonucleotides and spacer subunit(s), wherein: each spacer subunit consists of: (a) a phosphate or modified internucleoside linker and (b) a spacer; a spacer subunit can be at any position in the strand; the two strands form a duplex with at least one blunt end; and the 3 end of one or both strands terminates in a phosphate or modified internucleoside linker and further comprises, in 5 to 3 order: a second spacer; a second phosphate or modified internucleoside linker, and a 3 end cap. In various embodiments, the RNAi agents comprise a first and a second strand, wherein each strand is a 30-mer or shorter, the first strand comprises ribonucleotides, and the second strand comprises ribonucleotides and one or more spacer subunit(s), wherein: each spacer subunit consists of: (a) a phosphate or modified internucleoside linker and (b) a spacer; a spacer subunit can be at any position in the strand. In some embodiments, the 3 end of both strands further comprise, in 5 to 3 order: a second spacer; a second phosphate or modified internucleoside linker; and a 3 end cap. The two strands can have the same or different spacers, phosphates or modified internucleoside linkers, and/or 3 end caps. The strands can be ribonucleotides, or, optionally, one or more nucleotide can be modified or substituted. Optionally, at least one nucleotide comprises a modified internucleoside linker. Optionally, the first two base-pairing nucleotides on the 3 end of the one or both strand are 2-MOE. Optionally, the RNAi agent can be modified on one or both 5 end. Optionally, the first or second strand is the sense strand, and the sense strand can comprise a 5 end cap which reduces the amount of the RNA interference mediated by this strand. Optionally, the RNAi agent is attached to a ligand. This format can be used to devise RNAi agents to a variety of different targets and sequences. The disclosure also relates to processes for making such compositions, and methods and uses of such compositions, e.g., to mediate RNA interference.

    摘要翻译： 本公开涉及包含具有包括间隔子亚单位的新格式的RNAi剂的组合物。 本公开涉及包含具有新格式的RNAi剂的组合物：具有至少一个内部间隔物的18聚体形式。 这些RNAi剂包含第一和第二18聚体链，其中第一链是18个核糖核苷酸或18个总核糖核苷酸和间隔子亚单位，第二个链是18个总核糖核苷酸和间隔子亚基，其中： 间隔子亚基由以下组成：（a）磷酸酯或修饰的核苷间连接体和（b）间隔子; 间隔子亚基可以在链中的任何位置; 两条链与至少一个钝端形成双链体; 并且一条或两条链的3端终止于磷酸酯或修饰的核苷间连接体，并且还包含5至3个顺序：第二间隔物; 第二磷酸酯或修饰的核苷间连接体，和3端帽。 在各种实施方案中，RNAi试剂包含第一和第二链，其中每条链均为30聚体或更短，第一链包含核糖核苷酸，第二链包含核糖核苷酸和一个或多个间隔子亚单位，其中： 每个间隔子亚基由以下组成：（a）磷酸酯或修饰的核苷间连接体和（b）间隔子; 间隔子亚基可以在链中的任何位置。 在一些实施方案中，两条链的3端还包含5至3个顺序：第二间隔物; 第二磷酸酯或修饰的核苷间连接体; 和3个端盖。 两条链可以具有相同或不同的间隔物，磷酸酯或修饰的核苷间连接体和/或3个末端帽。 链可以是核糖核苷酸，或者任选地，一个或多个核苷酸可以被修饰或取代。 任选地，至少一个核苷酸包含修饰的核苷间连接体。 任选地，一条或两条链的3端上的前两个碱基配对核苷酸是2-MOE。 任选地，RNAi剂可在一端或两端进行修饰。 任选地，第一链或第二链是有义链，有义链可以包含5端帽，其减少由该链介导的RNA干扰的量。 任选地，RNAi试剂连接到配体上。 这种格式可用于将RNAi试剂设计成各种不同的靶标和序列。 本公开还涉及制备这种组合物的方法，以及这种组合物的方法和用途，例如介导RNA干扰。

    ORGANIC COMPOUNDS TO TREAT HEPATITIS B VIRUS
    6.
    发明申请
    ORGANIC COMPOUNDS TO TREAT HEPATITIS B VIRUS 审中-公开
    有机化合物治疗乙型肝炎病毒

    公开(公告)号：WO2015050871A2

    公开(公告)日：2015-04-09

    申请号：PCT/US2014058314

    申请日：2014-09-30

    申请人： NOVARTIS AG ,  BARYZA JEREMY LEE ,  BLOMMERS MARCEL ,  FERNANDEZ CESAR ,  GENO ERIN ,  GOSSERT ALVAR ,  GREENIDGE PAULETTE ,  HUESKEN DIETER ,  HUNZIKER JUERG ,  NATT FRANCOIS JEAN-CHARLES ,  PATNAIK ANUP ,  PATTERSON ANDREW ,  RONDEAU JEAN-MICHEL RENE ,  WEILER JAN ,  ZHU MEICHENG ,  HOLDORF MEGHAN

    发明人： BARYZA JEREMY LEE ,  BLOMMERS MARCEL ,  FERNANDEZ CESAR ,  GENO ERIN ,  GOSSERT ALVAR ,  GREENIDGE PAULETTE ,  HUESKEN DIETER ,  HUNZIKER JUERG ,  NATT FRANCOIS JEAN-CHARLES ,  PATNAIK ANUP ,  PATTERSON ANDREW ,  RONDEAU JEAN-MICHEL RENE ,  WEILER JAN ,  ZHU MEICHENG ,  HOLDORF MEGHAN

    IPC分类号： A61K48/00 ,  C07F9/6512 ,  C07H21/02

    CPC分类号： C12N15/1131 ,  C12N2310/14 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/332 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2320/30 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/3533

    摘要： The disclosure relates to compositions comprising a HBV RNAi agent. In some embodiments, the HBV RNAi agent comprises a sense and an anti-sense strand, each strand being an 18-mer and the strands together forming a blunt-ended duplex, wherein the 3' end of at least one strand terminates in a phosphate or modified internucleoside linker and further comprises, in 5' to 3' order: a spacer; a second phosphate or modified internucleoside linker; and a 3' end cap. In some embodiments, the 3' end of both the sense and anti-sense strand further comprise, in 5' to 3' order: a spacer; a second phosphate or modified internucleoside linker; and a 3' end cap. The two strands can have the same or different spacers, phosphates or modified internucleoside linkers, and/or 3' end caps. The strands can be ribonucleotides, or, optionally, one or more nucleotide can be modified or substituted. Optionally, at least one nucleotide comprises a modified internucleoside linker. Optionally, the RNAi agent can be modified on one or both 5' end. Optionally, the sense strand can comprise a 5' end cap which reduces the amount of the RNA interference mediated by this strand. Optionally, the RNAi agent is attached to a ligand. This format can be used to devise RNAi agents to a variety of different targets and sequences. The disclosure also relates to processes for making such compositions, and methods and uses of such compositions, e.g., to mediate RNA interference. The disclosure also pertains to methods of treating, ameliorating and preventing HBV in a patient involving the step of administering to the patient a therapeutic amount of a HBV RNAi agent.

    摘要翻译： 本公开涉及包含HBV RNAi剂的组合物。 在一些实施方案中，HBV RNAi剂包含有义链和反义链，每条链是18聚体，并且所述链一起形成平端双链体，其中至少一条链的3'末端终止于磷酸酯 或修饰的核苷间连接体，并且还包含以5'至3'的顺序：间隔物; 第二磷酸酯或修饰的核苷间连接体; 和3'端盖。 在一些实施方案中，有义和反义链两者的3'末端还包含5'至3'的顺序：间隔基; 第二磷酸酯或修饰的核苷间连接体; 和3'端盖。 两条链可以具有相同或不同的间隔物，磷酸酯或修饰的核苷间接头，和/或3'端帽。 链可以是核糖核苷酸，或者任选地，一个或多个核苷酸可以被修饰或取代。 任选地，至少一个核苷酸包含修饰的核苷间连接体。 任选地，RNAi剂可以在一个或两个5'端修饰。 任选地，有义链可以包含5'端帽，其减少由该链介导的RNA干扰的量。 任选地，RNAi试剂连接到配体上。 这种格式可用于将RNAi试剂设计成各种不同的靶标和序列。 本公开还涉及制备这种组合物的方法，以及这种组合物的方法和用途，例如介导RNA干扰。 本公开还涉及在患者中治疗，改善和预防HBV的方法，其涉及向患者施用治疗量的HBV RNAi剂的步骤。

    RNAI AGENTS TO TREAT BETA-CATENIN RELATED DISEASES
    8.
    发明申请
    RNAI AGENTS TO TREAT BETA-CATENIN RELATED DISEASES 审中-公开
    RNAI代理治疗β-卡培宁相关疾病

    公开(公告)号：WO2013105022A3

    公开(公告)日：2013-10-31

    申请号：PCT/IB2013050159

    申请日：2013-01-08

    申请人： NOVARTIS AG ,  HUESKEN DIETER

    发明人： HUESKEN DIETER ,  BETTENCOURT BRIAN ,  BUMCROT DAVID ,  KUCHIMANCHI SATYANARAYANA ,  MILSTEIN STUART ,  SCHLABACH JR MICHAEL ,  STEGMEIER FRANK P ,  WARMUTH MARKUS ,  WEILER JAN

    IPC分类号： C12N15/113 ,  A61K31/713

    CPC分类号： C12N15/113 ,  A61K31/713 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/344

    摘要： The present disclosure relates to RNAi agents useful in methods of treating Beta-Catenin-related diseases such as adenomatous polyposis of the colon, colorectal cancer, basal cell carcinoma, breast cancer, kidney cancer, Wilms tumors, medulloblastoma, ovarian cancer, adrenocortical tumors, gastric cancer, liver cancer, melanoma, pancreatic cancers, prostate cancer, renal cancer, ectopic teeth and taste papillae, skin cancer, pilomatrixoma, anaplastic thyroid carcinoma, and uterine carcinosarcoma, oligodontia, osteoporosis, ageing, degenerative diseases, bedsores, chronic wounds and impaired wound healing, and similar and related diseases, using a therapeutically effective amount of a RNAi agent to Beta-Catenin.

    摘要翻译： 本公开涉及可用于治疗β-联蛋白相关疾病如结肠腺瘤性息肉病，结肠直肠癌，基底细胞癌，乳腺癌，肾癌，Wilms肿瘤，成神经管细胞瘤，卵巢癌，肾上腺皮质肿瘤的方法中的RNAi剂， 胃癌，肝癌，黑素瘤，胰腺癌，前列腺癌，肾癌，异位牙齿和口腔乳头，皮肤癌，皮质细胞瘤，间变性甲状腺癌和子宫癌肉瘤，少痣，骨质疏松症，衰老，退行性疾病，褥疮，慢性伤口和 损伤的伤口愈合以及类似和相关疾病，使用治疗有效量的RNAi剂到β-联蛋白。

    ORGANIC COMPOSITIONS TO TREAT BETA-CATENIN-RELATED DISEASES
    9.
    发明申请
    ORGANIC COMPOSITIONS TO TREAT BETA-CATENIN-RELATED DISEASES 审中-公开
    有机组合物治疗β-卡培胺相关疾病

    公开(公告)号：WO2013105022A2

    公开(公告)日：2013-07-18

    申请号：PCT/IB2013/050159

    申请日：2013-01-08

    申请人： NOVARTIS AG ,  HUESKEN, Dieter

    发明人： HUESKEN, Dieter ,  BETTENCOURT, Brian ,  BUMCROT, David ,  KUCHIMANCHI, Satyanarayana ,  MILSTEIN, Stuart ,  SCHLABACH JR., Michael ,  STEGMEIER, Frank P. ,  WARMUTH, Markus ,  WEILER, Jan

    IPC分类号： C12N15/113 ,  A61K31/713

    CPC分类号： C12N15/113 ,  A61K31/713 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/344

    摘要： The present disclosure relates to RNAi agents useful in methods of treating Beta-Catenin-related diseases such as adenomatous polyposis of the colon, colorectal cancer, basal cell carcinoma, breast cancer, kidney cancer, Wilms tumors, medulloblastoma, ovarian cancer, adrenocortical tumors, gastric cancer, liver cancer, melanoma, pancreatic cancers, prostate cancer, renal cancer, ectopic teeth and taste papillae, skin cancer, pilomatrixoma, anaplastic thyroid carcinoma, and uterine carcinosarcoma, oligodontia, osteoporosis, ageing, degenerative diseases, bedsores, chronic wounds and impaired wound healing, and similar and related diseases, using a therapeutically effective amount of a RNAi agent to Beta-Catenin.

    摘要翻译： 本公开涉及可用于治疗β-联蛋白相关疾病如结肠腺瘤性息肉病，结肠直肠癌，基底细胞癌，乳腺癌，肾癌，Wilms肿瘤，成神经管细胞瘤，卵巢癌，肾上腺皮质肿瘤的方法中的RNAi剂， 胃癌，肝癌，黑素瘤，胰腺癌，前列腺癌，肾癌，异位牙齿和口腔乳头，皮肤癌，皮质细胞瘤，间变性甲状腺癌和子宫癌肉瘤，少痣，骨质疏松症，衰老，退行性疾病，褥疮，慢性伤口和 损伤的伤口愈合以及类似和相关疾病，使用治疗有效量的RNAi剂到β-联蛋白。