公开(公告)号：US20210246447A1

公开(公告)日：2021-08-12

申请号：US17121858

申请日：2020-12-15

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Akin Akinc ,  Gregory Hinkle ,  Martin A. Maier ,  James Butler ,  Jingxuan Liu

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K45/06 ,  A61K47/54 ,  C12Q1/6883

摘要： The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Kallikrein B, Plasma (Fletcher Factor) 1 (KLKB1) gene, the Factor XII (Hageman Factor (F12) gene, or the Kininogen 1 (KNG1) gene, and methods of using such RNAi agents to inhibit expression of a KLKB1 gene, an F12 gene, and/or a KNG1 gene, and methods of treating subjects having an hereditary angioedema (HAE) and/or a contact activation pathway-associated disorder.

公开(公告)号：US11053495B2

公开(公告)日：2021-07-06

申请号：US15745591

申请日：2016-07-15

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Vasant Jadhav ,  Martin Maier ,  Ivan Zlatev ,  Akin Akinc ,  Kallanthottathil G. Rajeev ,  Jayaprakash K. Nair ,  Pachamuthu Kandasamy ,  Muthiah Manoharan

IPC分类号： C12N15/113 ,  C12N15/11 ,  C07H21/00 ,  A61K47/55 ,  A61K47/54

摘要： The present invention relates, in general to, compounds, compositions and methods useful for modulating gene expression of multiple target nucleic acids by a single chemical entity.

公开(公告)号：US20210169915A1

公开(公告)日：2021-06-10

申请号：US16891155

申请日：2020-06-03

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Gregory Hinkle

IPC分类号： A61K31/712 ,  A61K31/7125 ,  A61P9/12 ,  A61K9/00 ,  C12N15/113 ,  A61K31/7115

摘要： The invention relates to polynucleotide agents, e.g., antisense polynucleotide agents, targeting an angiotensinogen (AGT) gene, and methods of using such polynucleotide agents to inhibit expression of AGT and to treat subjects having an AGT-associated disease, e.g., hypertension.

公开(公告)号：US11028392B2

公开(公告)日：2021-06-08

申请号：US16137046

申请日：2018-09-20

申请人： ALNYLAM PHARMACEUTICALS, INC. ,  ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI

发明人： Brian Bettencourt ,  Kevin Fitzgerald ,  William Querbes ,  Robert J. Desnick ,  Makiko Yasuda

IPC分类号： C12N15/113 ,  A61K9/00 ,  C12Q1/6876

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：US11015194B2

公开(公告)日：2021-05-25

申请号：US16042633

申请日：2018-07-23

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Muthiah Manoharan ,  Kallanthottathil G. Rajeev

IPC分类号： C12N15/113 ,  C12N15/11

摘要： The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent.

公开(公告)号：US20210108203A1

公开(公告)日：2021-04-15

申请号：US17128320

申请日：2020-12-21

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Kevin Fitzgerald ,  William Querbes ,  James Butler ,  Stephanie Williams ,  Abigail Liebow ,  Gregory Hinkle ,  Martin A. Maier ,  Stuart Milstein ,  Satyanarayana Kuchimanchi ,  Muthiah Manoharan

IPC分类号： C12N15/113

摘要： The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the apolipoprotein C3 (APOC3) gene, and methods of using such RNAi agents to inhibit expression of APOC3 and methods of treating subjects having an APOC3 associated disorder, e.g., hypertriglyceridemia.

公开(公告)号：US20210087558A1

公开(公告)日：2021-03-25

申请号：US16893253

申请日：2020-06-04

申请人： ALNYLAM PHARMACEUTICALS, INC. ,  ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI

发明人： Brian Bettencourt ,  Kevin Fitzgerald ,  William Querbes ,  Robert J. Desnick ,  Makiko Yasuda

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K47/60 ,  A61K48/00

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：US10935917B2

公开(公告)日：2021-03-02

申请号：US16513903

申请日：2019-07-17

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Alfica Sehgal ,  David Bumcrot ,  Brian Bettencourt

IPC分类号： A61K48/00 ,  C12N15/11 ,  G03G15/00 ,  C12N15/113 ,  G03G15/08

摘要： The technology described herein relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Serpina 1 gene, and methods of using such dsRNA compositions to inhibit expression of Serpina 1.

公开(公告)号：US20210002638A1

公开(公告)日：2021-01-07

申请号：US16939119

申请日：2020-07-27

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Kevin Fitzgerald ,  Huilei Xu ,  Gregory Hinkle

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K47/54

摘要： The invention relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the SCAP gene, as well as methods of inhibiting expression of a SCAP gene and methods of treating subjects having a SCAP-associated disorder, such as nonalcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH), using such dsRNAi agents and compositions.

公开(公告)号：US10767177B2

公开(公告)日：2020-09-08

申请号：US16002034

申请日：2018-06-07

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Kevin Fitzgerald ,  Huilei Xu ,  Gregory Hinkle

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K47/54

摘要： The invention relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the SCAP gene, as well as methods of inhibiting expression of a SCAP gene and methods of treating subjects having a SCAP-associated disorder, such as nonalcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH), using such dsRNAi agents and compositions.