- 专利标题: ANGIOPOIETIN-LIKE 3 (ANGLPTL3) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
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申请号: EP22168792.4申请日: 2012-06-20
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公开(公告)号: EP4092120A1公开(公告)日: 2022-11-23
- 发明人: Bettencourt, Brian , Querbes, William , Fitzgerald, Kevin , Frank-Kamenetsky, Maria , Milstein, Stuart , Shulga-Morskaya, Svetlana
- 申请人: Alnylam Pharmaceuticals, Inc.
- 申请人地址: US Cambridge, MA 02142 300 Third Street, 3rd Floor
- 代理机构: Vossius & Partner Patentanwälte Rechtsanwälte mbB
- 优先权: US201261638288 P 20120425
- 主分类号: C12N15/113
- 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.
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