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1.发明公开公开(公告)号：US20240309370A1
公开(公告)日：2024-09-19
申请号：US18403912
申请日：2024-01-04
发明人： Aimee M. Deaton , Jeffrey Zuber
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N15/1137 , C12N15/1138 , C12Y301/04017 , C12N2310/14 , C12N2310/3125 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/344 , C12N2310/351
摘要： The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting a metabolic disorder-associated target gene, e.g., inhibin subunit beta E (INHBE), activin A receptor type 1C (ACVR1C), perilipin-1 (PLIN1), phosphodiesterase 3B (PDE3B), or inhibin subunit beta C (INHBC) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a metabolic disorder-associated target gene and to methods of preventing and treating a metabolic disorder, e.g., metabolic syndrome.
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公开(公告)号：US20240301418A1
公开(公告)日：2024-09-12
申请号：US18572553
申请日：2022-06-23
IPC分类号： C12N15/113 , A61K31/713
CPC分类号： C12N15/113 , A61K31/713 , C12N2310/11 , C12N2310/315 , C12N2310/343 , C12N2310/3515 , C12N2310/352
摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a Filamin A (FLNA) gene, as well as methods of inhibiting expression of an FLNA gene and methods of treating subjects having an FLNA-associated N disease or disorder, e.g., Alzheimer's disease, using such dsRNAi agents and compositions.
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公开(公告)号：US20240294912A1
公开(公告)日：2024-09-05
申请号：US18519295
申请日：2023-11-27
发明人： Gregory Hinkle , Anna Borodovsky
IPC分类号： C12N15/113 , A61K31/7115
CPC分类号： C12N15/113 , A61K31/7115 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3235 , C12N2310/351
摘要： The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the complement factor C3 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of a C3 gene and to treat subjects having a complement component C3-associated disease, e.g., paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), atypical hemolytic uremic syndrome (aHUS), neuromyelitis optica (NMO), multifocal motor neuropathy (MMN), myasthenia gravis (MG), and C3 glomerulonephritis.
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公开(公告)号：US20240287524A1
公开(公告)日：2024-08-29
申请号：US18509583
申请日：2023-11-15
发明人： Kevin Fitzgerald , David Erbe , Gregory Hinkle
IPC分类号： C12N15/113 , A61K31/713 , A61K45/06
CPC分类号： C12N15/1137 , A61K31/713 , A61K45/06 , C12Y207/01002 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/351 , C12N2310/3521 , C12N2310/3533
摘要： The invention relates to double stranded ribonucleic acid (dsRNA) compositions targeting a glucokinase (GCK) gene, as well as methods of inhibiting expression of a glucokinase (GCK) gene, and methods of treating subjects having a glycogen storage disease (GSD), e.g., type Ia GSD.
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公开(公告)号：US20240285767A1
公开(公告)日：2024-08-29
申请号：US18589636
申请日：2024-02-28
发明人： Martin MAIER , Muthusamy JAYARAMAN , Akin AKINC , Shigeo MATSUDA , Pachamuthu KANDASAMY , Kallanthottathil G. RAJEEV , Muthiah MANOHARAN , Jayaprakash K. NAIR , Thomas A. BAILLIE
IPC分类号： A61K47/18 , A61K9/127 , A61K9/51 , A61K31/7088 , A61K31/7105 , A61K31/713 , C07C31/125 , C07C211/09 , C07C211/10 , C07C211/11 , C07C217/08 , C07C229/12 , C07C235/06 , C07C251/38 , C07C323/12 , C07C323/58 , C07C327/22 , C07C327/28 , C07C327/32 , C07D207/32 , C07D233/54 , C07D295/08 , C07D295/12 , C07D295/14 , C07D317/30 , C07F5/02
CPC分类号： A61K47/18 , A61K9/1272 , A61K9/5123 , A61K31/7088 , A61K31/7105 , A61K31/713 , C07C31/125 , C07C211/09 , C07C211/10 , C07C211/11 , C07C217/08 , C07C229/12 , C07C235/06 , C07C251/38 , C07C323/12 , C07C323/58 , C07C327/22 , C07C327/28 , C07C327/32 , C07D207/32 , C07D233/54 , C07D295/08 , C07D295/12 , C07D295/14 , C07D317/30 , C07F5/022
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：US20240254493A1
公开(公告)日：2024-08-01
申请号：US18562787
申请日：2022-05-27
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , C12Y402/01001 , C12N2310/14 , C12N2310/3125 , C12N2310/315 , C12N2310/3515
摘要： Carbonic anhydrase inhibitors have been shown to reduce aqueous humor production and thereby reduce intraocular pressure in the eye. Accordingly, there is a need for agents that can selectively and efficiently inhibit expression of the CA2 gene such that subjects having a CA2-associated disorder, such as glaucoma, can be effectively treated. The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting carbonic anhydrase 2 (CA2), and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of carbonic anhydrase 2.
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7.发明公开公开(公告)号：US20240254487A1
公开(公告)日：2024-08-01
申请号：US18532300
申请日：2023-12-07
发明人： JONATHAN EDWARD FARLEY , MARK K. SCHLEGEL , JAMES D. MCININCH , JEFFREY ZUBER , ADAM CASTORENO , STEPHEN ABBOTT , JOSEPH BARRY
IPC分类号： C12N15/113 , A61K48/00 , C12N15/85
CPC分类号： C12N15/113 , A61K48/005 , C12N15/85 , C12N2310/14 , C12N2310/315
摘要： The disclosure relates to double stranded ribonucleic acid interference (dsRNAi) agents and compositions targeting a microtubule-associated protein tau (MAPT) gene, as well as methods of inhibiting expression of a MAPT gene and methods of treating subjects having a MAPT-associated disease or disorder, e.g., Alzheimer's disease, frontotemporal dementia, progressive supranuclear palsy, or other tauopathies, using such dsRNAi agents and compositions.
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公开(公告)号：US20240252653A1
公开(公告)日：2024-08-01
申请号：US18441882
申请日：2024-02-14
发明人： Jayaprakash K. NAIR , Alexander V. KEL'IN , Pachamuthu KANDASAMY , Kallanthottathil G. RAJEEV , Muthiah MANOHARAN
IPC分类号： A61K47/54 , A61K31/7088 , C07C231/14 , C07D207/12 , C07H1/00 , C07H15/26 , C12N15/113
CPC分类号： A61K47/549 , A61K31/7088 , C07C231/14 , C07D207/12 , C07H1/00 , C07H15/26 , C12N15/113 , C12N2310/351 , C12N2320/32
摘要： This disclosure relates to an improved process for the preparation of carbohydrate conjugates. The disclosure also relates to carbohydrate conjugated iRNA agents comprising these carbohydrate conjugates, which have improved purity and are advantageous for the in vivo delivery of the iRNA agents.
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公开(公告)号：US20240247266A1
公开(公告)日：2024-07-25
申请号：US18542950
申请日：2023-12-18
发明人： Stephen Albert Huang , Kenji P. Fujita , Kang Lin , Nitin Mehrotra , Gabriel Robbie , Jongtae Lee , Sagar Agarwal
IPC分类号： C12N15/113 , A61K31/4184 , A61K31/713 , A61P9/12
CPC分类号： C12N15/1136 , A61K31/4184 , A61K31/713 , A61P9/12 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/323 , C12N2310/33 , C12N2310/351
摘要： The present invention relates to methods of inhibiting the expression of an AGT gene in a subject, as well as methods for treating subjects having an AGT-associated disorder, e.g., hypertension, using RNAi agents, e.g., double stranded RNAi agents, targeting the AGT gene. The invention also relates to methods of decreasing blood pressure levels in a subject using such RNAi agents to inhibit expression of an AGT gene.
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公开(公告)号：US20240218369A1
公开(公告)日：2024-07-04
申请号：US18385005
申请日：2023-10-30
发明人： John Vest
IPC分类号： C12N15/113 , A61P3/00 , A61P9/00 , A61P25/02 , A61P25/28
CPC分类号： C12N15/1136 , A61P3/00 , A61P9/00 , A61P25/02 , A61P25/28 , C12N2310/14
摘要： Disclosed herein are methods for treating hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in a human patient in need thereof by administering an effective amount of a transthyretin (TTR)-inhibiting composition.
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