-
公开(公告)号：US12049628B2
公开(公告)日：2024-07-30
申请号：US17584463
申请日：2022-01-26
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/322 , C12N2310/344 , C12N2310/351 , C12N2310/3515 , C12N2310/3533 , C12N2320/30 , C12N2310/322 , C12N2310/3533
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 diseases.
-
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.
-
公开(公告)号：US11590229B2
公开(公告)日：2023-02-28
申请号：US17651029
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.
-
公开(公告)号：US20220175929A1
公开(公告)日：2022-06-09
申请号：US17651029
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.
-
公开(公告)号：US11246933B1
公开(公告)日：2022-02-15
申请号：US17302311
申请日：2021-04-29
发明人： Martin Maier , Muthusamy Jayaraman , Akin Akinc , Shigeo Matsuda , Pachamuthu Kandasamy , Kallanthottathil G. Rajeev , Muthiah Manoharan
IPC分类号： A61K47/18 , A61K48/00 , 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.
-
公开(公告)号：US11060091B2
公开(公告)日：2021-07-13
申请号：US16673754
申请日：2019-11-04
发明人： Gregory Hinkle , Laura Sepp-Lorenzino , Vasant Jadhav , Martin Maier , Stuart Milstein , Muthiah Manoharan , Kallanthottathil G. Rajeev
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 B virus (HBV) genome, and methods of using such RNAi agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection and/or HBV-associated disorder, e.g., chronic hepatitis B infection.
-
公开(公告)号：US10995336B2
公开(公告)日：2021-05-04
申请号：US16420263
申请日：2019-05-23
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.
-
公开(公告)号：US10806791B2
公开(公告)日：2020-10-20
申请号：US16196628
申请日：2018-11-20
IPC分类号： A61K47/28 , A61K31/7004 , A61K31/7052 , A61K31/70 , A61K47/60 , A61K47/54 , C07H21/02 , A61K31/7088 , A61K31/713 , A61K47/16 , A61K47/22 , A61K48/00
摘要： The present invention provides a phosphorothioate-modified oligonucleotide comprising a structure shown below: The present invention also provides a phosphorothioate-modified oligonucleotide comprising a structure having formula (CIII):
-
9.发明申请公开(公告)号：US20200318111A1
公开(公告)日：2020-10-08
申请号：US16738014
申请日：2020-01-09
发明人： Kallanthottathil G. Rajeev , Tracy Zimmermann , Muthiah Manoharan , Martin A. Maier , Satyanarayana Kuchimanchi , Klaus Charisse
IPC分类号： C12N15/113 , A61K31/7125 , A61K31/713 , C07H21/02
摘要： 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.
-
公开(公告)号：US20200080085A1
公开(公告)日：2020-03-12
申请号：US16679691
申请日：2019-11-11
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 , A61K31/713 , A61K31/712
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.
-
-
-
-
-
-
-
-
-
搜索结果
127 条记录 (耗时 0.039 秒)
