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    ORGANIC COMPOUNDS TO TREAT HEPATITIS B VIRUS
    2.
    发明公开
    ORGANIC COMPOUNDS TO TREAT HEPATITIS B VIRUS 有权
    有机食品VERBINDUNGEN ZUR BEHANDLUNG VON HEPATITIS-B-INFEKTIONEN

    公开(公告)号：EP3052107A2

    公开(公告)日：2016-08-10

    申请号：EP14790848.7

    申请日：2014-09-30

    申请人： Novartis AG

    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： A61K31/713 ,  C12N15/113

    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 或修饰的核苷间连接体，并且还包含以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。 在一些实施方案中，有义链和反义链两者的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'端盖。 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。