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    NUCLEIC ACID INDUCING RNA INTERFERENCE MODIFIED FOR PREVENTING OFF-TARGET, AND USE THEREOF
    5.
    发明公开
    NUCLEIC ACID INDUCING RNA INTERFERENCE MODIFIED FOR PREVENTING OFF-TARGET, AND USE THEREOF 审中-公开
    RNA-INTERFERENZINDUZIERENDE MODIFIZIERTENUKLEINSÄLUREZUR VERHINDERUNG VON OFF-TARGET UND VERWENDUNG DAVON

    公开(公告)号：EP3085784A4

    公开(公告)日：2017-07-19

    申请号：EP14872036

    申请日：2014-12-08

    Applicant: ENCODEGEN CO LTD

    Inventor： CHI SUNG WOOK ,  JANG EUN-SOOK

    IPC: C12N15/113 ,  A61K48/00

    CPC classification number: C12N15/1137 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/31 ,  C12N2310/332 ,  C12N2310/344 ,  C12N2310/531 ,  C12N2310/533 ,  C12N2320/53 ,  C12N2310/321 ,  C12N2310/3521

    Abstract: The present invention relates to an RNA interference-inducing nucleic acid and the use thereof, and more particularly to an RNA interference-inducing nucleic acid comprising at least one single strand of double strands, the at least one single strand comprising a modification substituted to a spacer, which is unable to form a base pair, in the 5' end or the 3' end region. The RNA interference-inducing nucleic acid of the present invention is a novel modified form of nucleotide provided to prevent off-target effects, offering a method to selectively repress target gene expression. In addition, according to the present invention, the RNA interference-inducing nucleic acid provides novel modified forms with target selectivity and specificity as a method to block the off-target effects while silencing the target gene expression, whereas the usage of conventional RNA interference-inducing nucleic acids cause inaccuracy and adverse effects through off-targets, thereby the RNA interference-inducing nucleic acid was offered to solve the problem, wherein it will be widely used as a method for repressing gene expression in research and for gene therapy without concerning the off-target effects.

    Abstract translation: 本发明涉及诱导RNA干扰的核酸及其用途，并且更具体地涉及包含至少一条双链单链的RNA干扰诱导核酸，所述至少一条单链包含被取代成 在5'末端或3'末端区域中不能形成碱基对的间隔区。 本发明的RNA干扰诱导核酸是提供用于防止脱靶效应的新型修饰形式的核苷酸，提供选择性抑制靶基因表达的方法。 另外，根据本发明，诱导RNA干扰的核酸提供具有靶选择性和特异性的新型修饰形式作为在沉默靶基因表达的同时阻断脱靶效应的方法，而使用常规RNA干扰诱导型核酸， 诱导核酸通过脱靶导致不准确性和不良影响，因此提供了RNA干扰诱导核酸以解决该问题，其中它将被广泛用作在研究和基因治疗中抑制基因表达的方法，而不涉及 脱靶效应。

    NOVEL FORMATS FOR ORGANIC COMPOUNDS FOR USE IN RNA INTERFERENCE
    7.
    发明公开
    NOVEL FORMATS FOR ORGANIC COMPOUNDS FOR USE IN RNA INTERFERENCE 有权
    神经元FERR组织VERBINDUNGEN ZUR VERWENDUNG BEI RNA-INTERFERENZ

    公开(公告)号：EP3052627A2

    公开(公告)日：2016-08-10

    申请号：EP14792662.0

    申请日：2014-10-06

    Applicant: Novartis AG

    Inventor： 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: C12N15/113

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

    Abstract: 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.

    Abstract translation: 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基，第二链是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; 两条链形成具有至少一个钝端的双链体; 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，第一条链包含核糖核苷酸，第二条链包含核糖核苷酸和一个或多个间隔子亚基，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剂可以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。

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