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公开(公告)号：US20240294911A1
公开(公告)日：2024-09-05
申请号：US18509453
申请日：2023-11-15
发明人： James Butler , Kevin Fitzgerald , Gregory Hinkle , Brian Bettencourt , Huilei Xu
IPC分类号： C12N15/113 , A61P5/06
CPC分类号： C12N15/113 , A61P5/06 , C12N15/1136 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3341 , C12N2310/3515
摘要： The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the insulin-like growth factor binding protein, acid labile subunit (IGFALS) gene or the insulin-like growth factor 1 (IGF-1) gene, methods of using such double stranded RNAi agents to inhibit expression of an IGFALS gene or an IGF-1 gene, and methods of treating subjects having an IGF system-associated disorder.
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公开(公告)号：US11725209B2
公开(公告)日：2023-08-15
申请号：US17062748
申请日：2020-10-05
发明人： James Butler , Martin A. Maier , Kevin Fitzgerald , Shannon Fishman , Donald Foster , Vasant R. Jadhav , Stuart Milstein
IPC分类号： C07H21/04 , C07H21/02 , C12N15/113 , A61K31/713 , A61K45/06
CPC分类号： C12N15/1137 , A61K31/713 , A61K45/06 , C12N15/1138 , C12Y304/21 , C12N2310/14 , C12N2310/322 , C12N2310/351 , C12N2310/353 , C12N2310/3533 , C12N2320/31
摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as β-thalassemia or hemochromatosis.
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公开(公告)号：US20210163952A1
公开(公告)日：2021-06-03
申请号：US17062748
申请日：2020-10-05
发明人： James Butler , Martin A. Maier , Kevin Fitzgerald , Shannon Fishman , Donald Foster , Vasant R. Jadhav , Stuart Milstein
IPC分类号： C12N15/113 , A61K31/713 , A61K45/06
摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as β-thalassemia or hemochromatosis.
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公开(公告)号：US10913950B2
公开(公告)日：2021-02-09
申请号：US16191579
申请日：2018-11-15
发明人： James Butler , Brian Bettencourt , Kallanthottathil G. Rajeev , Martin A. Maier , Klaus Charisse
IPC分类号： A61K48/00 , C07H21/04 , C12N15/113 , C12N9/64
摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as β-thalassemia or hemochromatosis.
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公开(公告)号：US20200277599A1
公开(公告)日：2020-09-03
申请号：US16601681
申请日：2019-10-15
发明人： James Butler , Kevin Fitzgerald , Gregory Hinkle , Brian Bettencourt , Huilei Xu
IPC分类号： C12N15/113 , A61P5/06
摘要： The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the insulin-like growth factor binding protein, acid labile subunit (IGFALS) gene or the insulin-like growth factor 1 (IGF-1) gene, methods of using such double stranded RNAi agents to inhibit expression of an IGFALS gene or an IGF-1 gene, and methods of treating subjects having an IGF system-associated disorder.
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公开(公告)号：US20180087054A1
公开(公告)日：2018-03-29
申请号：US15782091
申请日：2017-10-12
发明人： William Querbes , Kevin Fitzgerald , James Butler , Stephanie Williams , Gregory Hinkle , Martin Maier
IPC分类号： C12N15/113 , A61K47/54 , A61K31/713
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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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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公开(公告)号：US20220267767A1
公开(公告)日：2022-08-25
申请号：US17487019
申请日：2021-09-28
发明人： James Butler , Jingxuan Liu
IPC分类号： C12N15/113 , A61P9/00
摘要： The present invention relates to methods of use of RNAi agents, e.g., double stranded RNAi agents, targeting a Factor XII (Hageman Factor (F12) gene, for treating subjects having a contact activation pathway-associated disease, such as a thrombophilia or hereditary angioedema (HAE), methods for preventing at least one symptom in a subject having a contact activation pathway-associated disease, such as a thrombus formation or an angioedema attack, and RNAi agents targeting an F12 gene, for use in the methods of the invention.
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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：US20200208150A1
公开(公告)日：2020-07-02
申请号：US16621754
申请日：2018-07-06
发明人： James Butler , Jingxuan Liu
IPC分类号： C12N15/113 , A61P9/00
摘要： The present invention relates to methods of use of RNAi agents, e.g., double stranded RNAi agents, targeting a Factor XII (Hageman Factor (F12) gene, for treating subjects having a contact activation pathway-associated disease, such as a thrombophilia or hereditary angioedema (HAE), methods for preventing at least one symptom in a subject having a contact activation pathway-associated disease, such as a thrombus formation or an angioedema attack, and RNAi agents targeting an F12 gene, for use in the methods of the invention.
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