公开(公告)号：EP3872179A1

公开(公告)日：2021-09-01

申请号：EP21150171.3

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： C12N15/113 ,  A61K31/713 ,  A61P3/06 ,  A61P9/00 ,  A61P43/00

摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the PCSK9 gene (PCSK9 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by PCSK9 gene expression and the expression of the PCSK9 gene using the pharmaceutical composition.

公开(公告)号：EP3578656A1

公开(公告)日：2019-12-11

申请号：EP19168142.8

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： C12N15/113 ,  A61K31/713

摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the PCSK9 gene (PCSK9 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by PCSK9 gene expression and the expression of the PCSK9 gene using the pharmaceutical composition.

公开(公告)号：EP3249052B1

公开(公告)日：2019-04-10

申请号：EP17162101.4

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： A61K31/713 ,  C12N15/113

公开(公告)号：EP3249052A1

公开(公告)日：2017-11-29

申请号：EP17162101.4

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 the PCSK9 gene (PCSK9 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by PCSK9 gene expression and the expression of the PCSK9 gene using the pharmaceutical composition.

摘要翻译： 本发明涉及用于抑制PCSK9基因（PCSK9基因）表达的双链核糖核酸（dsRNA），其包含具有长度小于30个核苷酸，通常长度为19-25个核苷酸的核苷酸序列的反义链 并且其与PCSK9基因的至少一部分基本上互补。 本发明还涉及包含dsRNA和药学上可接受的载体的药物组合物; 使用该药物组合物治疗由PCSK9基因表达引起的疾病和PCSK9基因表达的方法。