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17 条记录 (耗时 0.019 秒)

    RNAI AGENT TARGETING MARC1 GENE, AND USE THEREOF
    14.
    发明公开
    RNAI AGENT TARGETING MARC1 GENE, AND USE THEREOF 审中-实审

    公开(公告)号：EP4368717A1

    公开(公告)日：2024-05-15

    申请号：EP22838056.4

    申请日：2022-07-08

    申请人： Olix Pharmaceuticals, Inc.

    发明人： HONG, Sun Woo ,  PARK, June Hyun ,  CHOE, Jeong Yong

    IPC分类号： C12N15/113 ,  C07H19/048

    CPC分类号： C07H19/048 ,  C12N15/113

    摘要： The present disclosure relates to an RNAi agent targeting a mitochondrial amidoxime reducing component 1 (MARC1) gene and a use thereof, specifically, the present disclosure relates an RNAi agent including an antisense strand having sequence complementarity to a MARC1 mRNA sequence and a sense strand having sequence complementarity to the antisense strand, and a pharmaceutical composition including the RNAi agent for preventing or treating liver disease, such as non-alcoholic fatty liver disease.

    RNA-INTERFERENCE-INDUCING NUCLEIC ACID MOLECULE ABLE TO PENETRATE INTO CELLS, AND USE THEREFOR
    16.
    发明授权
    RNA-INTERFERENCE-INDUCING NUCLEIC ACID MOLECULE ABLE TO PENETRATE INTO CELLS, AND USE THEREFOR 审中-公开

    公开(公告)号：EP2853597B1

    公开(公告)日：2018-12-26

    申请号：EP13794539.0

    申请日：2013-05-21

    申请人： Olix Pharmaceuticals, Inc.

    发明人： HONG, Sun Woo

    IPC分类号： C12N15/113 ,  C12N15/63 ,  A61K48/00

    CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N15/87 ,  C12N2310/14 ,  C12N2310/313 ,  C12N2310/315 ,  C12N2310/3515 ,  C12N2320/32

    摘要： The present invention relates to a novel, RNAi-inducing nucleic acid molecule having cell penetrating ability and the use thereof, and more particularly, to a novel, RNAi-inducing double-stranded nucleic acid molecule, which has a replacement of the phosphate backbone of at least one nucleotide with phosphorothioate or phosphorodithioate, and has a lipophilic compound conjugated thereto, and thus has high target gene-silencing efficiency while having the ability to penetrate cells without needing a separate intracellular delivery vehicle, and to a method of silencing a target gene using the nucleic acid molecule. The nucleic acid structure according to the present invention has both cholesterol modification and phosphorothioate modification introduced therein, and thus has high gene silencing efficiency while having the ability to penetrate cells without needing a separate intracellular delivery vehicle. Thus, it can be delivered into an actual target area in an amount sufficient for induction of RNAi, and thus can overcome the in vivo delivery problem occurring in the prior art. Therefore, the nucleic acid molecule according to the invention can effectively substitute for conventional siRNA molecules to treat cancer or viral infections.