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公开(公告)号：US20220175930A1
公开(公告)日：2022-06-09
申请号：US17651038
申请日：2022-02-14
发明人： Martin Maier , Muthusamy Jayaraman , Akin Akinc , Shigeo Matsuda , Pachamuthu Kandasamy , Kallanthottathil G. Rajeev , Muthiah Manoharan
IPC分类号： A61K47/18 , A61K9/127 , A61K9/51 , A61K31/713 , C07D317/30 , C07C211/09 , C07C211/10 , C07C211/11 , C07C217/08 , C07C229/12 , C07C327/22 , C07C327/28 , C07C327/32 , C07C235/06 , C07C251/38 , C07F5/02 , C07D233/54 , C07D207/32 , C07D295/08 , C07D295/12 , C07D295/14 , C07C323/12 , C07C323/58 , C07C31/125 , A61K31/7088 , A61K31/7105
摘要： The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
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公开(公告)号：US20220175928A1
公开(公告)日：2022-06-09
申请号：US17651023
申请日：2022-02-14
发明人： Martin Maier , Muthusamy Jayaraman , Akin Akinc , Shigeo Matsuda , Pachamuthu Kandasamy , Kallanthottathil G. Rajeev , Muthiah Manoharan
IPC分类号： A61K47/18 , A61K9/127 , A61K9/51 , A61K31/713 , C07D317/30 , C07C211/09 , C07C211/10 , C07C211/11 , C07C217/08 , C07C229/12 , C07C327/22 , C07C327/28 , C07C327/32 , C07C235/06 , C07C251/38 , C07F5/02 , C07D233/54 , C07D207/32 , C07D295/08 , C07D295/12 , C07D295/14 , C07C323/12 , C07C323/58 , C07C31/125 , A61K31/7088 , A61K31/7105
摘要： The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
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公开(公告)号：US20220127615A1
公开(公告)日：2022-04-28
申请号：US17523030
申请日：2021-11-10
发明人： Akin Akinc , James D. McIninch , Yesseinia Anglero-Rodriguez , Mark K. Schlegel , Christy M. Hebner , Florian A. Lempp
IPC分类号： C12N15/113
摘要： The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the coronavirus genome. The invention also relates to methods of using such RNAi agents to inhibit expression of a coronavirus genome and to methods of treating or preventing a coronavirus-associated disease in a subject.
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公开(公告)号：US20220125927A1
公开(公告)日：2022-04-28
申请号：US17573878
申请日：2022-01-12
发明人： Martin Maier , Muthusamy Jayaraman , Akin Akinc , Shigeo Matsuda , Pachamuthu Kandasamy , Kallanthottathil G Rajeev , Muthiah Manoharan
IPC分类号： A61K47/18 , C07C323/58 , C07C327/28 , A61K31/713 , C07C323/12 , C07D233/54 , C07D295/12 , C07D295/14 , A61K9/51 , C07C31/125 , C07C211/09 , C07C235/06 , A61K31/7088 , C07C211/10 , C07C217/08 , C07D295/08 , C07D317/30 , A61K31/7105 , C07C327/22 , C07C327/32 , C07F5/02 , C07C211/11 , C07C251/38 , A61K9/127 , C07C229/12 , C07D207/32
摘要： The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
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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：US20170233734A1
公开(公告)日：2017-08-17
申请号：US15497943
申请日：2017-04-26
发明人： Akin Akinc , Martin Maier , Varun Kumar
IPC分类号： C12N15/113 , A61K9/127
CPC分类号： C12N15/113 , A61K9/0019 , A61K9/1272 , A61K9/1617 , A61K9/1641 , A61K31/713 , C12N15/88 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322
摘要： The present invention relates to lipid nanoparticles containing a biodegradable cationic lipid which provide improved delivery of active pharmaceutical ingredients, such as siRNA.
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公开(公告)号：US20160017330A1
公开(公告)日：2016-01-21
申请号：US14806084
申请日：2015-07-22
发明人： Akin Akinc , Alfica Sehgal , Ivanka Toudjarska , Donald Foster , Stuart Milstein , Brian Bettencourt , Martin Maier , Klaus Charisse , Satyanarayana Kuchimanchi , Kallanthottathil G. Rajeev , Muthiah Manoharan
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K31/713 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/335 , C12N2310/351 , C12N2310/3515 , C12N2310/3521 , C12N2310/3533 , C12N2320/30 , C12N2310/3525 , C12N2310/3523
摘要： The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and methods of treating subjects having a bleeding disorder, such as a hemophilia.
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公开(公告)号：US20240344066A1
公开(公告)日：2024-10-17
申请号：US18405072
申请日：2024-01-05
发明人： Akin Akinc , Jeffrey Zuber
IPC分类号： C12N15/113 , A61K45/06 , A61P35/00
CPC分类号： C12N15/113 , A61K45/06 , A61P35/00 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3515
摘要： The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the beta-catenin (CTNNB1) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a CTNNB1 gene and to methods of preventing and treating a CTNNB1-associated disorder, e.g., cancer, e.g., hepatocellular carcinoma.
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公开(公告)号：US20230287424A1
公开(公告)日：2023-09-14
申请号：US18077271
申请日：2022-12-08
发明人： Christopher Brown , Donald Foster , Arlin Rogers , Vasant R. Jadhav , Akin Akinc , Amy R. Simon
IPC分类号： C12N15/113 , A61K9/00
CPC分类号： C12N15/1131 , A61K9/0043 , C12N2310/14 , C12N2310/3515 , C12N2310/321 , C12N2310/315
摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents for administration by inhalation preferably targeting genes expressed in the respiratory system, as well as methods of inhibiting expression of a target gene after administration by inhalation, preferably a gene expressed in the respiratory system.
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10.发明公开公开(公告)号：US20230193284A1
公开(公告)日：2023-06-22
申请号：US17956884
申请日：2022-09-30
IPC分类号： C12N15/113 , C12N15/10
CPC分类号： C12N15/1138 , C12N15/1062 , C12Y304/21 , C12N2310/14 , C12N2310/315 , C12N2310/3515
摘要： The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the transmembrane serine protein 2 (TMPRSS2) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a TMPRSS2 gene and to methods of treating or preventing a TMPRSS2-associated disease, e.g., COVID-19, in a subject.
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