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公开(公告)号：US20180148722A1
公开(公告)日：2018-05-31
申请号：US15724175
申请日：2017-10-03
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： Kevin Fitzgerald , Maria Frank-Kamenetsky , Klaus Charisse
IPC: C12N15/113 , A61K31/713 , C07H21/02
CPC classification number: C12N15/113 , A61K31/713 , C07H21/02 , C12N15/1137 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/3519 , C12Y304/21061
Abstract: The invention relates to dual targeting siRNA agents targeting a PCSK9 gene and a second gene, and methods of using dual targeting siRNA agents to inhibit expression of PCSK9 and to treat PCSK9 related disorders, e.g., hyperlipidemia.
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公开(公告)号：US20170268005A1
公开(公告)日：2017-09-21
申请号：US15606224
申请日：2017-05-26
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： Kevin Fitzgerald , James Butler , Brian Bettencourt , Anna Borodovsky , Satyanarayana Kuchimanchi , Klaus Charisse , Muthiah Manoharan , Martin Maier , Kallanthottathil G. Rajeev , Donald Foster
IPC: C12N15/113 , A61K31/713 , C07K16/18 , A61K39/395
CPC classification number: C12N15/113 , A61K31/713 , A61K39/3955 , C07K16/18 , C07K2317/24 , C07K2317/76 , C12N2310/14 , C12N2310/3125 , C12N2310/315 , C12N2310/321 , C12N2310/351 , C12N2310/3527 , C12N2320/30
Abstract: The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.
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公开(公告)号：US09574192B2
公开(公告)日：2017-02-21
申请号：US14284745
申请日：2014-05-22
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： Alfica Sehgal , Klaus Charisse , Brian Bettencourt , Martin Maier , Kallanthottathil G. Rajeev , Gregory Hinkle , Muthiah Manoharan
IPC: C07H21/02 , C07H21/04 , A61K48/00 , C12N15/113 , A61K31/712 , A61K31/713
CPC classification number: C12N15/113 , A61K31/712 , A61K31/713 , C12N2310/11 , C12N2310/14 , C12N2310/315 , C12N2310/32 , C12N2310/321 , C12N2310/331 , C12N2310/332 , C12N2310/343 , C12N2310/344 , C12N2310/346 , C12N2310/351 , C12N2320/32 , C12N2320/34 , C12N2320/51 , C12N2330/30 , C12N2310/322 , C12N2310/3533 , C12N2310/3521
Abstract: The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.
Abstract translation: 本发明涉及靶向Serpina1基因的RNAi试剂，例如双链RNAi试剂，以及使用这种RNAi试剂抑制Serpina1表达的方法以及治疗患有Serpina1相关疾病如肝脏疾病的受试者的方法。
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24.发明申请RNAI AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING TRANSTHYRETIN (TTR) ASSOCIATED DISEASES 审中-公开RNAI代理，组合物及其治疗TRANSTHYRETIN（TTR）相关疾病的方法
公开(公告)号：US20160355817A1
公开(公告)日：2016-12-08
申请号：US15188317
申请日：2016-06-21
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： Kallanthottathil G. Rajeev , Tracy Zimmermann , Muthiah Manoharan , Martin Maier , Satyanarayana Kuchimanchi , Klaus Charisse
IPC: C12N15/113 , A61K31/713
Abstract: The present invention provides RNAi agents, e.g., double stranded RNAi agents, that target the transthyretin (TTR) gene and methods of using such RNAi agents for treating or preventing TTR-associated diseases.
Abstract translation: 本发明提供靶向转甲状腺素蛋白（TTR）基因的RNAi试剂，例如双链RNAi试剂，以及使用这种RNAi剂治疗或预防TTR相关疾病的方法。
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公开(公告)号：US20160312216A1
公开(公告)日：2016-10-27
申请号：US14885342
申请日：2015-10-16
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： Kevin Fitzgerald , Maria Frank-Kamenetsky , Klaus Charisse
IPC: C12N15/113
CPC classification number: C12N15/113 , A61K31/713 , C07H21/02 , C12N15/1137 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/3519 , C12Y304/21061
Abstract: The invention relates to dual targeting siRNA agents targeting a PCSK9 gene and a second gene, and methods of using dual targeting siRNA agents to inhibit expression of PCSK9 and to treat PCSK9 related disorders, e.g., hyperlipidemia.
Abstract translation: 本发明涉及靶向PCSK9基因和第二基因的双向靶向siRNA试剂，以及使用双重靶向siRNA试剂抑制PCSK9表达和治疗PCSK9相关病症例如高脂血症的方法。
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26.发明申请RNAI AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING TRANSTHYRETIN (TTR) ASSOCIATED DISEASES 有权RNAI代理，组合物及其治疗TRANSTHYRETIN（TTR）相关疾病的方法
公开(公告)号：US20140315835A1
公开(公告)日：2014-10-23
申请号：US14358972
申请日：2012-11-16
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： Kallanthottathil G. Rajeev , Tracy Zimmermann , Muthiah Manoharan , Martin Maier , Satyanarayana Kuchimanchi , Klaus Charisse
IPC: C12N15/113
CPC classification number: C12N15/113 , A61K31/7125 , A61K31/713 , C07H21/02 , C12N15/1136 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/343 , C12N2310/346 , C12N2310/351 , C12N2320/30 , C12N2310/3521 , C12N2310/3533
Abstract: The present invention provides RNAi agents, e.g., double stranded RNAi agents, that target the transthyretin (TTR) gene and methods of using such RNAi agents for treating or preventing TTR-associated diseases.
Abstract translation: 本发明提供靶向转甲状腺素蛋白（TTR）基因的RNAi试剂，例如双链RNAi试剂，以及使用这种RNAi剂治疗或预防TTR相关疾病的方法。
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27.发明公开
公开(公告)号：US20240279652A1
公开(公告)日：2024-08-22
申请号：US18509440
申请日：2023-11-15
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： Kallanthottathil G. Rajeev , Tracy Zimmermann , Muthiah Manoharan , Martin A. Maier , Satyanarayana Kuchimanchi , Klaus Charisse
IPC: C12N15/113 , A61K31/7125 , A61K31/713 , C07H21/02
CPC classification number: C12N15/113 , A61K31/7125 , A61K31/713 , C07H21/02 , C12N15/1136 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/343 , C12N2310/346 , C12N2310/351 , C12N2320/30
Abstract: The present invention provides RNAi agents, e.g., double stranded RNAi agents, that target the transthyretin (TTR) gene and methods of using such RNAi agents for treating or preventing TTR-associated diseases.
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28.发明公开
公开(公告)号：US20240110180A1
公开(公告)日：2024-04-04
申请号：US18199390
申请日：2023-05-19
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： Brian Bettencourt , Kevin Fitzgerald , Stuart Milstein , Martin A. Maier , Klaus Charisse , Kallanthottathil G. Rajeev , Satyanarayana Kuchimanchi , Muthiah Manoharan , Tuyen M. Nguyen
IPC: C12N15/113
CPC classification number: C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/344 , C12N2310/3515 , C12N2310/3521
Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) targeting an APOC3 gene, and methods of using the dsRNA to inhibit expression of APOC3.
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公开(公告)号：US11725207B2
公开(公告)日：2023-08-15
申请号：US17194431
申请日：2021-03-08
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： Mark K. Schlegel , Maja Janas , Vasant R. Jadhav , Donald Foster , Muthiah Manoharan , Kallanthottathil G. Rajeev , Alexander V. Kel'in , Klaus Charisse , Jayaprakash K. Nair , Martin A. Maier , Shigeo Matsuda , Muthusamy Jayaraman , Alfica Sehgal , Christopher Brown , Kevin Fitzgerald , Stuart Milstein
IPC: C12N15/113
CPC classification number: C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/351
Abstract: The invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.
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公开(公告)号：US20230123192A1
公开(公告)日：2023-04-20
申请号：US17855851
申请日：2022-07-01
Applicant: Alnylam Pharmaceuticals, Inc.
Inventor： Donald Foster , Brian Bettencourt , Klaus Charisse , Gregory Hinkle , Satyanarayana Kuchimanchi , Martin A. Maier , Stuart Milstein
IPC: A61K47/54 , C12N15/113 , A61K45/06
Abstract: The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the angiotensinogen (AGT) gene, and methods of using such RNAi agents to inhibit expression of AGT and methods of treating subjects having an AGT-associated disorder, e.g., hypertension.
