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公开(公告)号：US11413348B2
公开(公告)日：2022-08-16
申请号：US16741100
申请日：2020-01-13
IPC分类号： A61K47/18 , C07C211/08 , C07C211/04 , C07C211/17 , C07C211/21 , C12N15/88 , C07D207/06 , A61K48/00 , A61K45/00
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
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公开(公告)号：US09896688B2
公开(公告)日：2018-02-20
申请号：US14769361
申请日：2014-02-20
发明人： Wonsuk Chang , Erin N. Guidry , Matthew G. Stanton , Daniel Zewge
CPC分类号： C12N15/113 , C07H19/10 , C07H21/00 , C07H21/02 , C07H21/04 , C12N15/111 , C12N2310/11 , C12N2310/14 , C12N2310/319
摘要： The present invention relates to RNAi molecules, and compositions thereof, comprising a 2′ internucleoside linkage connecting the nucleotide at position 1 and the nucleotide at position 2 at the 5′ end of the antisense strand. Specifically, the invention relates to single- and double-stranded short interfering nucleic acid (siNA) molecules that are capable of mediating RNA interference comprising 5′ modified nucleotides that comprise, among other potential modifications, a 2′ internucleoside linkage. The invention further relates to 5′ modified nucleotides used as reagents to generate the RNAi molecules of the invention and methods of using the disclosed RNAi molecules.
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公开(公告)号：US09850491B2
公开(公告)日：2017-12-26
申请号：US15224822
申请日：2016-08-01
发明人： Duncan Brown , James J. Cunningham , Marian Gindy , Victoria Pickering , Matthew G. Stanton , Steven M. Stirdivant , Walter R. Strapps
IPC分类号： A61K48/00 , C12N15/11 , C07H21/02 , C07H21/04 , C12N15/113
CPC分类号： C12N15/1138 , C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/332 , C12N2310/346 , C12N2310/3521 , C12N2320/32
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of CTNNB1 gene expression and/or activity, and/or modulate a beta-catenin gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that medium RNA interference (RNAi) against CTNNB1 gene expression.
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公开(公告)号：US09642804B2
公开(公告)日：2017-05-09
申请号：US14876402
申请日：2015-10-06
发明人： Mark Cameron , Jennifer R. Davis , Andrea R. Geiser , Matthew G. Stanton , Vladislav V. Telyatnikov , Lu Tian , Weimin Wang
IPC分类号： A61K9/00 , C07J41/00 , C07J43/00 , A61K9/127 , A61K31/7088
CPC分类号： A61K9/1272 , A61K31/7088 , C07J41/0055 , C07J43/003
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with siRNA, to facilitate the cellular uptake and endosomal escape, and to knockdown target mRNA both in vitro and in vivo.
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公开(公告)号：US09458090B2
公开(公告)日：2016-10-04
申请号：US14682494
申请日：2015-04-09
IPC分类号： A61K9/51 , C07C229/12 , C12N15/88 , A61K48/00
CPC分类号： C07C229/12 , A61K9/5123 , A61K31/221 , A61K48/00 , C12N15/88
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids comprising at least one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
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6.发明申请NOVEL DIESTER AND TRIESTER BASED LOW MOLECULAR WEIGHT, BIODEGRADEABLE CATIONIC LIPIDS FOR OLIGONUCLEOTIDE DELIVERY 有权新型柴油机和基于TRIESTER的低分子量，用于寡核苷酸交付的生物降解性阳离子脂肪公开(公告)号：US20150133519A1
公开(公告)日：2015-05-14
申请号：US14395267
申请日：2013-04-16
IPC分类号： A61K9/51 , C12N15/113 , C07C229/28 , C07C229/30
CPC分类号： A61K9/5123 , A61K9/51 , A61K9/5146 , A61K31/713 , A61K47/60 , C07C229/12 , C07C229/28 , C07C229/30 , C07C2101/02 , C07C2601/02 , C07F7/1804 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2320/32 , C12N2320/51
摘要： The instant invention provides for novel cationic lipids of Formula A that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain coupled with inclusion of hydrolysable functionality in the lipid chains to enhance the efficiency and tolerability of in vivo delivery of siRNA.
摘要翻译： 本发明提供式A的新型阳离子脂质，其可以与其它脂质组分如胆固醇和PEG-脂质组合使用以与寡核苷酸形成脂质纳米颗粒。 本发明的目的是提供一种阳离子脂质支架，由于肝中脂质水平降低，其表现出增强的功效以及较低的肝毒性。 本发明使用具有一个短脂链的低分子量阳离子脂质，并在脂质链中加入可水解官能团，以提高siRNA体内递送的效率和耐受性。
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7.发明申请公开(公告)号：US20220195433A1
公开(公告)日：2022-06-23
申请号：US17510277
申请日：2021-10-25
发明人： Mark Cancilla , James John Cunningham , William Michael Flanagan , Henry J. Haringsma , Denise M. Kenski , Matthew G. Stanton , Steven M. Stirdivant , Aarron T. Willingham
IPC分类号： C12N15/113 , C12N15/11 , A61K31/713 , C07H21/02
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (MAO against target gene expression.
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8.发明授权公开(公告)号：US11193126B2
公开(公告)日：2021-12-07
申请号：US15947248
申请日：2018-04-06
发明人： Mark Cancilla , James John Cunningham , William Michael Flanagan , Henry J. Haringsma , Denise M. Kenski , Matthew G. Stanton , Steven M. Stirdivant , Aarron T. Willingham
IPC分类号： C07H21/02 , C07H21/04 , C12N15/113 , C12N15/11 , A61K31/713
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.
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公开(公告)号：US09643916B2
公开(公告)日：2017-05-09
申请号：US15077447
申请日：2016-03-22
发明人： Brian W. Budzik , Steven L. Colletti , Jennifer R. Davis , Ivory D. Hills , Darla Danile Seifried , Matthew G. Stanton
IPC分类号： C07C217/46 , C07D317/28 , C07C217/44 , C07D295/084
CPC分类号： C07C217/46 , A61K9/1272 , A61K47/18 , C07C43/15 , C07C217/44 , C07D205/04 , C07D207/08 , C07D295/084 , C07D317/28 , C12N15/87
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
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10.发明授权RNA interference mediated inhibition of catenin (cadherin-associated protein), beta 1 (CTNNB1) gene expression using short interfering nucleic acid (SINA) 有权使用短干扰核酸（SINA）的RNA干扰介导的连环素（钙粘蛋白相关蛋白），β1（CTNNB1）基因表达的抑制公开(公告)号：US09447420B2
公开(公告)日：2016-09-20
申请号：US14339067
申请日：2014-07-23
发明人： Duncan Brown , James J. Cunningham , Marian Gindy , Victoria Pickering , Matthew G. Stanton , Steven M. Stirdivant , Walter R. Strapps
IPC分类号： C12N15/11 , C07H21/02 , C07H21/04 , A61K48/00 , C12N15/113
CPC分类号： C12N15/1138 , C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/332 , C12N2310/346 , C12N2310/3521 , C12N2320/32
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of CTNNB1 gene expression and/or activity, and/or modulate a beta-catenin gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against CTNNB1 gene expression.
摘要翻译： 本发明涉及用于研究，诊断和治疗对CTNNB1基因表达和/或活性的调节作用和/或调节β-连环蛋白基因表达途径的性状，疾病和病症的化合物，组合物和方法 。 具体地说，本发明涉及包括小核酸分子，如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）等双链核酸分子， 和能够介导或介导针对CTNNB1基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子。
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