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    Compositions and methods for inhibiting expression of the PCSK9 gene
    1.
    发明公开
    Compositions and methods for inhibiting expression of the PCSK9 gene 有权
    用于抑制PCSK9基因表达的组合物和方法

    公开(公告)号：EP2584048A1

    公开(公告)日：2013-04-24

    申请号：EP12178667.7

    申请日：2007-05-10

    申请人： Alnylam Pharmaceuticals Inc.

    发明人： Tan, Pamela ,  Bramlage, Birgit ,  Frank-Kamenetsky, Maria ,  Fitzgerald, Kevin ,  Akinc, Akin ,  Kotelianski, Victor, E.

    IPC分类号： C12Q1/68 ,  A01N43/04 ,  C07H21/04 ,  A61K31/07 ,  C12N15/113

    CPC分类号： C12N15/1137 ,  A61K31/713 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/332 ,  C12N2310/3515 ,  C12N2310/3521

    摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a human PCSK9 gene in a cell, wherein said dsRNA comprises at least two sequences that are complementary to each other and wherein a sense strand comprises a first sequence and an antisense strand comprises a second sequence comprising a region of complementarity which is substantially complementary to at least a part of a mRNA encoding PCSK9, and wherein said region of complementarity is less than 30 nucleotides in length and wherein said dsRNA, upon contact with a cell expressing said PCSK9, inhibits expression of said PCSK9 gene, wherein (a) said first sequence is the sequence of SEQ ID NO: 1229 and said second sequence is the sequence of SEQ ID NO: 1230; (b) said first sequence is the sequence of SEQ ID NO: 1227 and said second sequence is the sequence of SEQ ID NO: 1228; or (c) said part of said mRNA encoding PCSK9 is the sequence from position 3530 to 3548 of the mRNA sequence of database entry NM_174936.

    摘要翻译： 本发明涉及用于抑制细胞中人PCSK9基因表达的双链核糖核酸（dsRNA），其中所述dsRNA包含至少两个彼此互补的序列，并且其中有义链包含第一序列和 反义链包含第二序列，所述第二序列包含与编码PCSK9的mRNA的至少一部分基本互补的互补区域，并且其中所述互补区域的长度小于30个核苷酸，并且其中所述dsRNA在与细胞接触时 其中（a）所述第一序列是SEQ ID NO：1229的序列，并且所述第二序列是SEQ ID NO：1230的序列;和（b）表达所述PCSK9抑制所述PCSK9基因的表达。 （b）所述第一序列是SEQ ID NO：1227的序列，并且所述第二序列是SEQ ID NO：1228的序列; 或（c）编码PCSK9的所述mRNA的所述部分是数据库条目NM_174936的mRNA序列的位置3530至3548的序列。

    ANGIOPOIETIN-LIKE 3 (ANGLPTL3) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
    4.
    发明公开
    ANGIOPOIETIN-LIKE 3 (ANGLPTL3) IRNA COMPOSITIONS AND METHODS OF USE THEREOF 审中-公开

    公开(公告)号：EP4092120A1

    公开(公告)日：2022-11-23

    申请号：EP22168792.4

    申请日：2012-06-20

    申请人： Alnylam Pharmaceuticals, Inc.

    发明人： Bettencourt, Brian ,  Querbes, William ,  Fitzgerald, Kevin ,  Frank-Kamenetsky, Maria ,  Milstein, Stuart ,  Shulga-Morskaya, Svetlana

    IPC分类号： C12N15/113 ,  A61K31/713 ,  C07H21/02

    摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) agent for inhibiting expression of Angiopoietin-like 3 (ANGPTL3), comprising a sense strand and an antisense strand forming a duplex region, wherein the sense strand comprises at least 15, preferably at least 16 and most preferably at least 17 contiguous nucleotides of the nucleotide sequence 5' -GUCUCAAAAUGGAAGGUUAUA- 3 ' (SEQ ID NO: 1160) , wherein the antisense strand comprises at least 15 contiguous nucleotides of the nucleotide sequence 5' -UAUAACCUUCCAUUUUGAGACUU-3' (SEQ ID NO: 1341) , wherein at least one of the modified nucleotides is selected from the group consisting of a 2'-O- methyl modified nucleotide, a 2 '-fluoro modified nucleotide, and a nucleotide comprising a 5'-phosphorothioate group, and wherein a ligand comprising an N-acetylgalactosamine (GaINAc) is conjugated to at least one strand of the dsRNA agent.