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

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： TAN, Pamela ,  BRAMLAGE, Birgit ,  FRANK-KAMENETSKY, Maria ,  FITZGERALD, Kevin ,  AKINC, Akin ,  KOTELIANSKI, Victor E.

IPC: 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

Applicant: Alnylam Pharmaceuticals, Inc.

Inventor： TAN, Pamela ,  BRAMLAGE, Birgit ,  FRANK-KAMENETSKY, Maria ,  FITZGERALD, Kevin ,  AKINC, Akin ,  KOTELIANSKI, Victor E.

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

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

Abstract: 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.

Abstract translation: 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。

公开(公告)号：EP2331690B1

公开(公告)日：2016-01-13

申请号：EP09792177.9

申请日：2009-09-02

Applicant: Alnylam Pharmaceuticals Inc. ,  Alnylam Europe AG. ,  The Regents of the University of California

Inventor： SAH, Dinah ,  TAN, Pamela ,  PEREZ, Maria Del Mar Inda ,  FURNARI, Frank ,  CAVENEE, Webster ,  BONAVIA, Rudy

IPC: C12N15/113 ,  A61K31/713

CPC classification number: C12N15/1138 ,  A61K31/713 ,  C12N15/1136 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3515 ,  C12N2320/30 ,  C12N2320/31 ,  C12N2310/3521

Abstract: The invention relates to combinations of a double-stranded ribonucleic acid (dsRNA) targeting a mutant Epidermal Growth Factor Receptor (EGFR), and a double-stranded ribonucleic acid (dsRNA) targeting interleukin-6 (IL-6), and their use in treating cancer.

公开(公告)号：EP2013222B1

公开(公告)日：2013-02-13

申请号：EP07761452.7

申请日：2007-04-27

Applicant: Alnylam Pharmaceuticals Inc.

Inventor： TAN, Pamela ,  SAH, Dinah ,  BRAMLAGE, Birgit

IPC: C07H21/04

CPC classification number: C12N15/1131 ,  A61K48/005 ,  C07H21/02 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3521

公开(公告)号：EP1942948A2

公开(公告)日：2008-07-16

申请号：EP06837005.5

申请日：2006-11-03

Applicant: Alnylam Pharmaceuticals Inc.

Inventor： SAH, Dinah, Wen-Yee ,  FRANK-KAMENETSKY, Maria ,  GEICK, Anke ,  HADWIGER, Philipp ,  ROEHL, Ingo ,  TAN, Pamela ,  VORNLOCHER, Hans-Peter

IPC: A61K48/00 ,  C07J17/00 ,  C07H21/02 ,  C12N5/10 ,  C12N5/08 ,  C07F9/6533

CPC classification number: C12N15/1138 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3515 ,  C12N2310/3521

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Nav1.8 gene (Nav1.8 gene), comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Nav1.8 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the Nav1.8 gene using the pharmaceutical composition; and methods for inhibiting the expression of the Nav1.8 gene gene in a cell

公开(公告)号：EP2023937B1

公开(公告)日：2011-10-12

申请号：EP07797577.9

申请日：2007-05-18

Applicant: Alnylam Pharmaceuticals Inc. ,  The Scripps Research Institute

Inventor： CONSTIEN, Rainer ,  BRAMLAGE, Birgit ,  TAN, Pamela ,  VORNLOCHER, Hans-Peter ,  BALCH, William

IPC: A61K31/70 ,  C12N15/11

CPC classification number: C12N15/113 ,  C12N2310/14 ,  C12N2310/3515

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of an Aha gene (Aha1 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 an Aha gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Aha1 expression and the expression of an Aha gene using the pharmaceutical composition; and methods for inhibiting the expression of an Aha gene in a cell.

公开(公告)号：EP2137313A2

公开(公告)日：2009-12-30

申请号：EP08718165.7

申请日：2008-03-25

Applicant: Novartis AG ,  Alnylam Pharmaceuticals, Inc

Inventor： BENSON, John ,  FITZGERALD, Kevin ,  BRAMLAGE, Birgit ,  TAN, Pamela ,  VORNLOCHER, Hans-Peter

IPC: C12N15/11

CPC classification number: C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3515 ,  C12Y603/02019 ,  C12N2310/3521

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for treating human papilloma virus (HPV) infection. The dsRNA comprises 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 an HPV Target gene selected from the human E6AP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by HPV infection and the expression of the E6AP gene using the pharmaceutical composition; and methods for inhibiting the expression of the HPV Target genes in a cell.

公开(公告)号：EP2069380A2

公开(公告)日：2009-06-17

申请号：EP07842674.9

申请日：2007-09-18

Applicant: Alnylam Pharmaceuticals Inc. ,  Board of Regents, The University of Texas System

Inventor： SOUTSCHEK, Juergen ,  TAN, Pamela ,  HORTON, Jay, D. ,  BROWN, Michael, S. ,  GOLDSTEIN, Joseph, L. ,  MOON, Young-ah

IPC: C07H21/04 ,  C12N15/00

CPC classification number: C12N15/113 ,  C12N2310/14 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/346 ,  C12N2310/3515 ,  C12N2310/3521

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a SCAP gene (Human SCAP 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 a SCAP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Human SCAP expression and the expression of a SCAP gene using the pharmaceutical composition; and methods for inhibiting the expression of a SCAP gene in a cell.

公开(公告)号：EP2013222A2

公开(公告)日：2009-01-14

申请号：EP07761452.7

申请日：2007-04-27

Applicant: Alnylam Pharmaceuticals Inc.

Inventor： TAN, Pamela ,  SAH, Dinah ,  BRAMLAGE, Birgit

IPC: C07H21/04

CPC classification number: C12N15/1131 ,  A61K48/005 ,  C07H21/02 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3521

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a gene from the JC Virus (JC virus genome), 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 a gene from the JC Virus. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by JC virus expression and the expression of a gene from the JC Virus using the pharmaceutical composition; and methods for inhibiting the expression of a gene from the JC Virus in a cell.

Abstract translation: 本发明涉及用于抑制来自JC病毒（JC病毒基因组）的基因表达的双链核糖核酸（dsRNA），其包含具有长度小于30个核苷酸的核苷酸序列的反义链， 长度为25个核苷酸，并且与来自JC病毒的基因的至少一部分基本互补。 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; 使用药物组合物治疗由JC病毒表达引起的疾病和来自JC病毒的基因表达的方法; 以及抑制细胞中来自JC病毒的基因表达的方法。

公开(公告)号：EP1941059A2

公开(公告)日：2008-07-09

申请号：EP06827135.2

申请日：2006-10-27

Applicant: Alnylam Pharmaceuticals Inc.

Inventor： SAH, Dinah Wen-Yee ,  HADWIGER, Philipp ,  ROEHL, Ingo ,  BRAMLAGE, Birgit ,  TAN, Pamela ,  VORNLOCHER, Hans-Peter ,  BUMCROT, David

IPC: C12Q1/68 ,  A01N43/04 ,  C07H21/04 ,  A61K31/07

CPC classification number: C12N15/113 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3515 ,  C12N2310/3521

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