公开(公告)号：EP3284824A1

公开(公告)日：2018-02-21

申请号：EP17175148.0

申请日：2013-04-10

申请人： Alnylam Pharmaceuticals, Inc. ,  Icahn School of Medicine at Mount Sinai

发明人： BETTENCOURT, Brian ,  FITZGERALD, Kevin ,  QUERBES, William ,  YASUDA, Makiko ,  DESNICK, Robert J.

IPC分类号： C12N15/113 ,  A61K31/713

CPC分类号： C12N15/113 ,  A61K31/713 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/343 ,  C12N2310/344 ,  C12N2310/351 ,  C12N2310/3521 ,  C12N2310/3533 ,  C12N2310/50 ,  C12N2310/533 ,  C12N2320/30 ,  C12Y203/01037

摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.

公开(公告)号：EP2836595B1

公开(公告)日：2017-06-14

申请号：EP13721433.4

申请日：2013-04-10

申请人： Alnylam Pharmaceuticals, Inc. ,  Icahn School of Medicine at Mount Sinai

发明人： BETTENCOURT, Brian ,  FITZGERALD, Kevin ,  QUERBES, William ,  YASUDA, Makiko ,  DESNICK, Robert, J.

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61P43/00

CPC分类号： C12N15/113 ,  A61K31/713 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/343 ,  C12N2310/344 ,  C12N2310/351 ,  C12N2310/3521 ,  C12N2310/3533 ,  C12N2310/50 ,  C12N2310/533 ,  C12N2320/30 ,  C12Y203/01037

摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.

摘要翻译： 本发明涉及靶向ALAS1基因的双链核糖核酸（dsRNA）组合物，以及使用这种dsRNA组合物改变（例如抑制）ALAS1表达的方法。

公开(公告)号：EP3052628A1

公开(公告)日：2016-08-10

申请号：EP14800179.5

申请日：2014-10-03

申请人： Alnylam Pharmaceuticals, Inc. ,  Icahn School of Medicine at Mount Sinai

发明人： BETTENCOURT, Brian ,  FITZGERALD, Kevin ,  QUERBES, William ,  DESNICK, Robert, J. ,  YASUDA, Makiko

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K31/713 ,  A61P3/00

摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.

公开(公告)号：EP3868883A1

公开(公告)日：2021-08-25

申请号：EP21155819.2

申请日：2013-04-10

申请人： Alnylam Pharmaceuticals, Inc. ,  Icahn School of Medicine at Mount Sinai

发明人： BETTENCOURT, Brian ,  FITZGERALD, Kevin ,  QUERBES, William ,  YASUDA, Makiko ,  DESNICK, Robert J.

IPC分类号： C12N15/113 ,  A61K31/713

摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting expression of ALAS1, wherein the dsRNA comprises a sense strand and an antisense strand, the antisense strand comprising a region of complementarity to an ALAS1 transcript, which antisense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from the antisense sequence of SEQ ID NO: 3553.

公开(公告)号：EP3052628B1

公开(公告)日：2020-02-26

申请号：EP14800179.5

申请日：2014-10-03

申请人： Alnylam Pharmaceuticals, Inc. ,  Icahn School of Medicine at Mount Sinai

发明人： BETTENCOURT, Brian ,  FITZGERALD, Kevin ,  QUERBES, William ,  DESNICK, Robert, J. ,  YASUDA, Makiko

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K31/713 ,  A61P3/00

公开(公告)号：EP2836595A2

公开(公告)日：2015-02-18

申请号：EP13721433.4

申请日：2013-04-10

申请人： Alnylam Pharmaceuticals, Inc. ,  Icahn School of Medicine at Mount Sinai

发明人： BETTENCOURT, Brian ,  FITZGERALD, Kevin ,  QUERBES, William ,  YASUDA, Makiko ,  DESNICK, Robert, J.

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61P43/00

CPC分类号： C12N15/113 ,  A61K31/713 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/343 ,  C12N2310/344 ,  C12N2310/351 ,  C12N2310/3521 ,  C12N2310/3533 ,  C12N2310/50 ,  C12N2310/533 ,  C12N2320/30 ,  C12Y203/01037

摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.

公开(公告)号：EP4376953A1

公开(公告)日：2024-06-05

申请号：EP22850557.4

申请日：2022-07-29

申请人： Alnylam Pharmaceuticals, Inc.

发明人： NAIR, Jayaprakash, K. ,  PANDYA, Bhaumik, A. ,  LENTINI, Scott, P. ,  MALONE, Ryan ,  PENG, Haiyan ,  CASTELLANOS-RIZALDOS, Elena ,  THEILE, Christopher, S. ,  MATSUDA, Shigeo ,  MAIER, Martin, A. ,  JADHAV, Vasant, R. ,  FITZGERALD, Kevin ,  TOLENTINO, Mark, Neil ,  ZLATEV, Ivan

IPC分类号： A61P25/16 ,  C07H21/02 ,  C12N15/113

CPC分类号： C12N15/113 ,  A61P25/16 ,  C12N2310/32120130101 ,  C12N2310/32220130101 ,  C12N2310/1420130101 ,  C12N2310/351320130101 ,  C12N2320/3220130101 ,  C12N15/1137 ,  C12N2310/31520130101 ,  C12N2310/34320130101 ,  A61K47/64 ,  C07K19/00 ,  A61K47/65

公开(公告)号：EP3983077A1

公开(公告)日：2022-04-20

申请号：EP20826062.0

申请日：2020-06-16

申请人： Alnylam Pharmaceuticals, Inc.

发明人： NAIR, Jayaprakash K. ,  MAIER, Martin ,  JADHAV, Vasant ,  MILSTEIN, Stuart ,  BROWN, Kirk ,  RAJEEV, Kallanthottathil G. ,  MANOHARAN, Muthiah ,  KEL'IN, Alexander V. ,  JAYARAMAN, Muthusamy ,  CHARISSE, Klaus ,  CASTORENO, Adam ,  THEILE, Christopher S. ,  FITZGERALD, Kevin ,  PARMAR, Rubina G.

IPC分类号： A61P25/28 ,  C12N15/113

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：EP3256587A2

公开(公告)日：2017-12-20

申请号：EP16706299.1

申请日：2016-02-11

申请人： Alnylam Pharmaceuticals, Inc.

发明人： FITZGERALD, Kevin ,  HINKLE, Gregory

IPC分类号： C12N15/113 ,  A61K31/713

CPC分类号： A61K31/7125 ,  C07H19/067 ,  C07H19/167 ,  C07H21/02 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3515 ,  C12P19/34 ,  C12N2310/3521 ,  C12N2310/3533

摘要： The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene, and methods of using such RNAi agents to inhibit expression of a PNPLA3 gene and methods of treating subjects having Nonalcoholic Fatty Liver Disease (NAFLD) and/or a PNPLA3-associated disorder.