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公开(公告)号：US20240200075A1
公开(公告)日：2024-06-20
申请号：US18337495
申请日：2023-06-20
发明人： William Querbes , Kevin Fitzgerald , James Butler , Stephanie Williams , Gregory Hinkle , Martin A. Maier
IPC分类号： C12N15/113 , A61K31/713 , A61K47/54
CPC分类号： C12N15/1136 , A61K31/713 , A61K47/549 , C12N2310/14 , C12N2310/315 , C12N2310/34 , C12N2310/344 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/32 , C12N2320/35 , C12N2320/51
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.
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2.发明公开公开(公告)号：US20240200061A1
公开(公告)日：2024-06-20
申请号：US18225919
申请日：2023-07-25
发明人： Jayaprakash K. Nair , Martin A. Maier , Vasant R. Jadhav , Mark Keating , Kevin Fitzgerald , Stuart Milstein , Kirk Brown , Muthiah Manoharan
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2320/34
摘要： The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the transthyretin (TTR) gene and methods of using such iRNA agents for treating or preventing TTR-associated ocular diseases.
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3.发明公开公开(公告)号：US20230304002A1
公开(公告)日：2023-09-28
申请号：US17736251
申请日：2022-05-04
发明人： Jayaprakash K. Nair , Martin A. Maier , Vasant R. Jadhav , Mark Keating , Kevin Fitzgerald , Stuart Milstein , John R. Petrulis
IPC分类号： C12N15/113 , A61K47/54
CPC分类号： C12N15/113 , A61K47/543 , A61K47/549 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3513 , C12N2310/3515 , C12N2320/32
摘要： The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the transthyretin (TTR) gene and methods of using such iRNA agents for treating or preventing TTR-associated ocular diseases.
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公开(公告)号：US20230002772A1
公开(公告)日：2023-01-05
申请号：US17586850
申请日：2022-01-28
发明人： Anna Borodovsky , Kallanthottathil G. Rajeev , Kevin Fitzgerald , Maria Frank-Kamenetsky , William Querbes , Martin A. Maier , Klaus Charisse , Satyanarayana Kuchimanchi , Muthiah Manoharan , Stuart Milstein
IPC分类号： C12N15/113 , A61K31/713 , A61K47/54
摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the PCSK9 gene, and methods of using such RNAi agents to inhibit expression of PCSK9 and methods of treating subjects having a lipid disorder, such as a hyperlipidemia.
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公开(公告)号：US20210317452A1
公开(公告)日：2021-10-14
申请号：US17141275
申请日：2021-01-05
发明人： Alfica Sehgal , Klaus Charisse , Brian Bettencourt , Martin A. Maier , Kallanthottathil G. Rajeev , Gregory Hinkle , Muthiah Manoharan
IPC分类号： C12N15/113 , A61K31/712 , A61K31/713
摘要： 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：US20210269796A1
公开(公告)日：2021-09-02
申请号：US17194431
申请日：2021-03-08
发明人： 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分类号： C12N15/113
摘要： 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：US10934544B2
公开(公告)日：2021-03-02
申请号：US16421745
申请日：2019-05-24
发明人： Akin Akinc , Gregory Hinkle , Martin A. Maier , James Butler , Jingxuan Liu
IPC分类号： C12N15/11 , C07H21/02 , C07H21/04 , 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.
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公开(公告)号：US20200291405A1
公开(公告)日：2020-09-17
申请号：US16801373
申请日：2020-02-26
发明人： Gregory Hinkle , Laura Sepp-Lorenzino , Vasant R. Jadhav , Martin A. Maier , Muthiah Manoharan , Stuart Milstein , Svetlana Shulga Morskaya , Kallanthottathil G. Rajeev , Huilei Xu
IPC分类号： C12N15/113 , A61K31/713 , A61K45/06
摘要： The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the hepatitis D virus (HDV) genome, and methods of using such RNAi agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HDV infection and/or HDV-associated disorder.
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9.发明申请公开(公告)号：US20180362978A1
公开(公告)日：2018-12-20
申请号：US15948624
申请日：2018-04-09
发明人： Brian Bettencourt , Kevin Fitzgerald , Stuart Milstein , Martin A. Maier , Klaus Charisse , Kallanthottathil Rajeev , Satyanarayana Kuchimanchi , Muthiah Manoharan , Tuyen Nguyen
IPC分类号： C12N15/113
摘要： 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：US11873491B2
公开(公告)日：2024-01-16
申请号：US17372710
申请日：2021-07-12
发明人： Kevin Fitzgerald , James Butler , Brian Bettencourt , Anna Borodovsky , Satyanarayana Kuchimanchi , Klaus Charisse , Muthiah Manoharan , Martin A. Maier , Kallanthottathil G. Rajeev , Donald Foster
IPC分类号： C12N15/113 , A61K31/713 , A61K39/395 , C07K16/18
CPC分类号： C12N15/113 , A61K31/713 , A61K39/3955 , C07K16/18 , C07K2317/24 , C07K2317/76 , C12N2310/14 , C12N2310/315 , C12N2310/3125 , C12N2310/321 , C12N2310/351 , C12N2310/3527 , C12N2320/30
摘要： 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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