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    iRNA agents with biocleavable tethers
    42.
    发明授权
    iRNA agents with biocleavable tethers 有权
    具有生物可清除系绳的iRNA试剂

    公开(公告)号：US08426377B2

    公开(公告)日：2013-04-23

    申请号：US12724267

    申请日：2010-03-15

    申请人： Muthiah Manoharan ,  Kallanthottathil G. Rajeev

    发明人： Muthiah Manoharan ,  Kallanthottathil G. Rajeev

    IPC分类号： A61K31/70 ,  C07H21/04 ,  C07H5/06

    CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/323 ,  C12N2310/3511 ,  C12N2320/51

    摘要： The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent.

    摘要翻译： 本发明涉及iRNA试剂，其优选包括其中核糖部分已被核糖替代的单体，其进一步包括具有一个或多个连接基团的系链，其中至少一个连接基团是可切割的 连接组。 系链又可以连接到所选择的部分，例如配体，例如靶向或递送部分，或改变物理性质的部分。 可切割连接基团是在细胞外部足够稳定的连接基团，使得其允许靶向治疗有益量的iRNA试剂（例如，单链或双链iRNA试剂），其通过可切割连接基团连接至 靶向细胞靶向细胞，但是在进入靶细胞时被切割以从靶向剂释放iRNA试剂。

    Oligonucleotides comprising a non-phosphate backbone linkage
    48.
    发明授权
    Oligonucleotides comprising a non-phosphate backbone linkage 有权
    包含非磷酸骨架键的寡核苷酸

    公开(公告)号：US08013136B2

    公开(公告)日：2011-09-06

    申请号：US12496268

    申请日：2009-07-01

    申请人： Muthiah Manoharan ,  Kallanthottathil G. Rajeev

    发明人： Muthiah Manoharan ,  Kallanthottathil G. Rajeev

    IPC分类号： C07H21/02 ,  C07H21/04

    CPC分类号： C12N15/111 ,  C07H19/04 ,  C07H19/12 ,  C07H19/16 ,  C07H19/167 ,  C07H19/173 ,  C07H21/02 ,  C12N15/1136 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/312 ,  C12N2320/51

    摘要： One aspect of the present invention relates to a ribonucleoside substituted with a phosphonamidite group at the 3′-position. In certain embodiments, the phosphonamidite is an alkyl phosphonamidite. Another aspect of the present invention relates to a double-stranded oligonucleotide comprising at least one non-phosphate linkage. Representative non-phosphate linkages include phosphonate, hydroxylamine, hydroxylhydrazinyl, amide, and carbamate linkages. In certain embodiments, the non-phosphate linkage is a phosphonate linkage. In certain embodiments, a non-phosphate linkage occurs in only one strand. In certain embodiments, a non-phosphate linkage occurs in both strands. In certain embodiments, a ligand is bound to one of the oligonucleotide strands comprising the double-stranded oligonucleotide. In certain embodiments, a ligand is bound to both of the oligonucleotide strands comprising the double-stranded oligonucleotide. In certain embodiments, the oligonucleotide strands comprise at least one modified sugar moiety. Another aspect of the present invention relates to a single-stranded oligonucleotide comprising at least one non-phosphate linkage. Representative non-phosphate linkages include phosphonate, hydroxylamine, hydroxylhydrazinyl, amide, and carbamate linkages. In certain embodiments, the non-phosphate linkage is a phosphonate linkage. In certain embodiments, a ligand is bound to the oligonucleotide strand. In certain embodiments, the oligonucleotide comprises at least one modified sugar moiety.

    摘要翻译： 本发明的一个方面涉及在3'-位被亚磷酰胺基取代的核糖核苷。 在某些实施方案中，亚磷酰胺是烷基亚磷酰胺。 本发明的另一方面涉及包含至少一种非磷酸键的双链寡核苷酸。 代表性的非磷酸酯键包括膦酸酯，羟胺，羟基肼基，酰胺和氨基甲酸酯键。 在某些实施方案中，非磷酸酯键是膦酸酯键。 在某些实施方案中，非磷酸酯键仅出现在一条链中。 在某些实施方案中，在两条链中都发生非磷酸连接。 在某些实施方案中，配体与包含双链寡核苷酸的寡核苷酸链之一结合。 在某些实施方案中，配体与包含双链寡核苷酸的两条寡核苷酸链结合。 在某些实施方案中，寡核苷酸链包含至少一个修饰的糖部分。 本发明的另一方面涉及包含至少一种非磷酸键的单链寡核苷酸。 代表性的非磷酸酯键包括膦酸酯，羟胺，羟基肼基，酰胺和氨基甲酸酯键。 在某些实施方案中，非磷酸酯键是膦酸酯键。 在某些实施方案中，配体与寡核苷酸链结合。 在某些实施方案中，寡核苷酸包含至少一个修饰的糖部分。