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1.发明申请公开(公告)号：US20200377889A1
公开(公告)日：2020-12-03
申请号：US16864226
申请日：2020-05-01
IPC分类号： C12N15/113
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.
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公开(公告)号：US20200308588A1
公开(公告)日：2020-10-01
申请号：US16954625
申请日：2018-12-18
IPC分类号： C12N15/113
摘要： The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the HMGB 1 gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a HMGB1 gene and to methods of N preventing and treating an HMGB1-associated disorder, e.g., metabolic disorder or non-alcholic fatty liver disease, e.g., non-alcoholic steatohepatitis (NASH).
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公开(公告)号：US20240336914A1
公开(公告)日：2024-10-10
申请号：US18259887
申请日：2021-12-30
发明人： Shigeo MATSUDA , Jayaprakash K. NAIR , Martin A. MAIER , Michelle H. JUNG , Anna BISBE , Ivan ZLATEV , Mark K. SCHLEGEL , Christopher BROWN , Vasant R. JADHAV
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/351
摘要： This invention relates to an oligonucleotide comprising one or more 2′-modified nucleosides, wherein the 2′-position of the nucleoside has a structure of formula (I). The invention also relates to a pharmaceutical composition comprising the oligonucleotide described herein and a method of reducing or inhibiting the expression of a target gene by administering to the subject a therapeutically effective amount of the oligonucleotide described herein. The invention also relates to a method of bioactivating an oligonucleotide comprising one or more 2′-modified nucleosides, wherein the 2′-position of the nucleoside is modified by a bio-cleavable linking group, wherein the bio-cleavable linking group comprises acetal, disulfide, carbamate, amide, sulfonamide, a biocleavable carbohydrate linker, or combinations thereof, said method comprising the step of: exposing the oligonucleotide to a physiological condition that causes the bio-cleavable linking group to be cleaved from the 2′-modified nucleoside, thereby regenerating the 2′-OH group of the nucleoside.
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4.发明公开公开(公告)号：US20240327834A1
公开(公告)日：2024-10-03
申请号：US18390233
申请日：2023-12-20
IPC分类号： C12N15/113 , A61K31/713
CPC分类号： C12N15/113 , A61K31/713 , C12N2310/11 , C12N2310/14 , C12N2310/321 , C12N2310/344 , C12N2310/3515
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a Serum Amyloid A (SAA) gene, and methods of using the dsRNA to inhibit expression of SAA.
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公开(公告)号：US20240285768A1
公开(公告)日：2024-08-29
申请号：US18594172
申请日：2024-03-04
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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6.发明公开公开(公告)号：US20240279652A1
公开(公告)日：2024-08-22
申请号：US18509440
申请日：2023-11-15
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
CPC分类号： C12N15/113 , A61K31/7125 , A61K31/713 , C07H21/02 , C12N15/1136 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/343 , C12N2310/346 , C12N2310/351 , C12N2320/30
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.
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公开(公告)号：US12059468B2
公开(公告)日：2024-08-13
申请号：US17368246
申请日：2021-07-06
IPC分类号： A61K31/7004 , A61K31/70 , A61K31/7052 , A61K31/7088 , A61K31/713 , A61K47/16 , A61K47/22 , A61K47/28 , A61K47/54 , A61K47/60 , C07H21/02 , A61K48/00
CPC分类号： A61K47/28 , A61K31/70 , A61K31/7004 , A61K31/7052 , A61K31/7088 , A61K31/713 , A61K47/16 , A61K47/22 , A61K47/543 , A61K47/549 , A61K47/60 , C07H21/02 , A61K48/00 , Y02A50/30 , Y02P20/55
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):-
公开(公告)号：US20240254491A1
公开(公告)日：2024-08-01
申请号：US18581511
申请日：2024-02-20
发明人： Akin Akinc , Jeffrey Zuber
IPC分类号： C12N15/113 , A61K31/713 , A61K47/14 , A61K47/18 , A61K47/28
CPC分类号： C12N15/113 , A61K31/713 , A61K47/14 , A61K47/18 , A61K47/28 , C12N2310/14 , C12N2310/312 , C12N2310/315 , C12N2310/321 , C12N2310/322
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：US20240218366A1
公开(公告)日：2024-07-04
申请号：US18586689
申请日：2024-02-26
IPC分类号： C12N15/113 , A61K9/00 , A61K45/06 , A61K47/54 , A61P7/02
CPC分类号： C12N15/113 , A61K9/0019 , A61K45/06 , A61K47/549 , A61P7/02 , C12N2310/11 , C12N2310/3125 , C12N2310/313
摘要： The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the Coagulation Factor V (F5) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an F5 gene and to methods of treating or preventing an F5-associated disease, e.g., a disorder associated with thrombosis, in a subject.
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公开(公告)号：US20240200077A1
公开(公告)日：2024-06-20
申请号：US18550669
申请日：2022-03-17
IPC分类号： C12N15/113 , A61M5/142
CPC分类号： C12N15/1137 , A61M5/14212 , C12N2310/14 , C12N2310/3515
摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a Stearoyl-CoA Desaturase 5 (SCD5) gene, as well as methods of inhibiting expression of an SCD5 gene and methods of treating subjects having an SCD5-associated disease or disorder, e.g., Parkinson's disease, using such dsRNAi agents and compositions.
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