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公开(公告)号:US09512164B2
公开(公告)日:2016-12-06
申请号:US13382353
申请日:2010-07-07
IPC分类号: C12N5/02 , C12N15/113 , C07H21/02 , C07H21/04
CPC分类号: C12N15/113 , C07H21/02 , C07H21/04 , C12N2310/14 , C12N2310/344
摘要: Modified nucleic acids are described herein, including pharmaceutical compositions comprising the modified nucleic acids, and methods of using the modified nucleic acids.
摘要翻译: 本文描述了修饰的核酸,包括包含修饰的核酸的药物组合物,以及使用经修饰的核酸的方法。
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公开(公告)号:US08927513B2
公开(公告)日:2015-01-06
申请号:US13382346
申请日:2010-07-07
IPC分类号: A01N43/04 , C07H19/11 , C07H19/213
CPC分类号: C12N15/113 , C07H19/10 , C07H19/11 , C07H19/20 , C07H19/213 , C12N2310/11
摘要: The present invention provides nucleosides and oligonucleotides comprising a 5′ phosphate mimics of formula (IVc) or (Vc). One aspect of the present invention relates to modified nucleosides and oligonucleotides comprising such dinucleotide of formula (Ia). Another aspect of the invention relates to a method of inhibiting the expression of a gene in call, the method comprising (a) contacting an oligonucleotide of the invention with the cell; and (b) maintaining the cell from step (a) for a time sufficient to obtain degradation of the mRNA of the target gene.
摘要翻译: 本发明提供了包含式(IVc)或(Vc)的5'磷酸酯模拟物的核苷和寡核苷酸。 本发明的一个方面涉及包含式(Ia)的二核苷酸的修饰核苷和寡核苷酸。 本发明的另一方面涉及一种抑制基因表达的方法,所述方法包括(a)使本发明的寡核苷酸与细胞接触; 和(b)将来自步骤(a)的细胞维持足以获得靶基因的mRNA降解的时间。
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公开(公告)号:US20130317080A1
公开(公告)日:2013-11-28
申请号:US13822441
申请日:2011-09-14
申请人: Kallanthottathil G. Rajeev , Tracy Zimmermann , Muthiah Manoharan , Martin Maier , Kevin Fitzgerald
发明人: Kallanthottathil G. Rajeev , Tracy Zimmermann , Muthiah Manoharan , Martin Maier , Kevin Fitzgerald
IPC分类号: C12N15/113
CPC分类号: C12N15/113 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/322 , C12N2310/343 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/32 , C12N2320/52 , C12N2310/3533
摘要: The present invention provides effective motifs for RNA agents conjugated to at least one ligand, which are advantageous for the in vivo delivery of iRNA duplex agents. Additionally, the present invention provides methods of making these compositions, as well as methods of introducing these iRNA duplex agents into cells using these compositions, e.g., for the treatment of various disease conditions.
摘要翻译: 本发明提供了与至少一种配体缀合的RNA试剂的有效基序,其有利于iRNA双链体的体内递送。 此外,本发明提供了制备这些组合物的方法,以及使用这些组合物将这些iRNA双链体引入细胞的方法,例如用于治疗各种疾病状况。
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公开(公告)号:US20130202652A1
公开(公告)日:2013-08-08
申请号:US13813365
申请日:2011-07-29
CPC分类号: A61K47/44 , A61K47/183 , A61K47/42 , A61K47/54 , A61K47/542 , A61K47/549 , A61K47/60 , A61K47/61 , A61K47/62 , A61K47/6911 , A61K47/6919
摘要: A targeting lipid can have the formula (I) in which T4 is a targeting moiety. The targeting moiety can be chosen to favor the lipid being localized with a desired organ, tissue, cell, cell type or subtype, or organelle. The targeting moiety can include, for example, transferrin, anisamide, an RGD peptide, prostate specific membrane antigen (PSMA), fucose, an antibody, or an aptamer.
摘要翻译: 靶向脂质可以具有式(I),其中T4是靶向部分。 可以选择靶向部分以有利于所需的器官,组织,细胞,细胞类型或亚型或细胞器定位脂质。 靶向部分可以包括例如转铁蛋白,茴香酰胺,RGD肽,前列腺特异性膜抗原(PSMA),岩藻糖,抗体或适体。
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公开(公告)号:US20130129785A1
公开(公告)日:2013-05-23
申请号:US13697229
申请日:2011-05-10
IPC分类号: A61K47/18 , C07C229/12 , C07C233/38 , A61K31/7088 , C07C271/12 , C07C275/20 , C07C271/64 , C07C219/08 , C07C237/06
CPC分类号: A61K47/18 , A61K9/1272 , A61K9/1278 , A61K31/7088 , C07C219/08 , C07C229/12 , C07C233/38 , C07C237/06 , C07C271/12 , C07C271/64 , C07C275/20 , C12N15/88
摘要: A lipid particle can include a cationic lipid. Synthesis of the cationic lipid can include a ylide-based reaction, such as a Wittig reaction or sulfur ylide reaction. In some cases, the synthesis can also include a Michael addition or a related addition reaction.
摘要翻译: 脂质颗粒可以包括阳离子脂质。 阳离子脂质的合成可以包括基于叶立德的反应,例如Wittig反应或硫叶立德反应。 在某些情况下,合成还可以包括迈克尔加成或相关的加成反应。
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公开(公告)号:US08404862B2
公开(公告)日:2013-03-26
申请号:US12497262
申请日:2009-07-02
IPC分类号: C07F9/06
CPC分类号: C12N15/113 , A61K31/7088 , A61K31/713 , A61K47/54 , A61K47/543 , A61K47/551 , A61K47/554 , C07F9/06 , C12N15/111 , C12N15/87 , C12N2310/113 , C12N2310/14 , C12N2310/351 , C12N2310/3515 , C12N2320/32 , C12N2320/51
摘要: This invention relates composition and methods for making and using chemically modified oligonucleotides agents for inhibiting gene expression.
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公开(公告)号:US20130018085A1
公开(公告)日:2013-01-17
申请号:US13620212
申请日:2012-09-14
申请人: Antonin de Fougerolles , Maria Frank-Kamenetsky , Muthiah Manoharan , Kallanthottathil G. Rajeev , Philipp Hadwiger
发明人: Antonin de Fougerolles , Maria Frank-Kamenetsky , Muthiah Manoharan , Kallanthottathil G. Rajeev , Philipp Hadwiger
IPC分类号: A61K31/713 , A61P35/00 , A61P27/02 , C12N5/071 , C07H21/02
CPC分类号: C12N15/1136 , A61K9/0019 , A61K31/7088 , A61K31/712 , A61K31/713 , A61K47/02 , A61K47/10 , A61K47/12 , A61K47/549 , A61K47/551 , A61K47/60 , A61K48/00 , C07K14/475 , C12N2310/14 , C12N2310/3515 , C12N2310/533 , C12N2320/30 , C12N2320/32
摘要: The features of the present invention relate to compounds, compositions and methods useful for modulating the expression of vascular endothelial growth factor (VEGF), such as by the mechanism of RNA interference (RNAi). The compounds and compositions include iRNA agents that can be unmodified or chemically-modified.
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公开(公告)号:US08344125B2
公开(公告)日:2013-01-01
申请号:US12619382
申请日:2009-11-16
CPC分类号: C12N15/111 , A61K47/554 , C12N2310/14 , C12N2310/323 , C12N2310/344 , C12N2310/3515 , C12N2320/51
摘要: The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose. The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated, e.g., by using the non-ribose moiety as a point to which a ligand or other entity, e.g., a carbohydrate; or a steroid, e.g., cholesterol, which is optionally substituted with at least one carbohydrate. is directly, or indirectly, tethered. The invention also relates to methods of making and using such modified iRNA agents.
摘要翻译: 本发明涉及iRNA试剂,其优选包括其中核糖部分被除核糖以外的部分替代的单体。 包含这样的单体可以允许调节其引入其中的iRNA试剂的性质,例如通过使用非核糖部分作为配体或其他实体例如碳水化合物的点; 或类固醇,例如胆固醇,其任选被至少一种碳水化合物取代。 是直接或间接地束缚着。 本发明还涉及制备和使用这种修饰的iRNA试剂的方法。
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公开(公告)号:US20120157511A1
公开(公告)日:2012-06-21
申请号:US13382346
申请日:2010-07-07
IPC分类号: A61K31/7088 , C07H19/11 , C12N5/071 , C07H19/073 , C07H21/00 , C07H21/02 , C07H19/04 , C07H19/10
CPC分类号: C12N15/113 , C07H19/10 , C07H19/11 , C07H19/20 , C07H19/213 , C12N2310/11
摘要: The present invention provides nucleosides and oligonucleotides comprising a 5′ phosphate mimics of formula (IVc) or (Vc). One aspect of the present invention relates to modified nucleosides and oligonucleotides comprising such dinucleotide of formula (Ia). Another aspect of the invention relates to a method of inhibiting the expression of a gene in call, the method comprising (a) contacting an oligonucleotide of the invention with the cell; and (b) maintaining the cell from step (a) for a time sufficient to obtain degradation of the mRNA of the target gene.
摘要翻译: 本发明提供了包含式(IVc)或(Vc)的5'磷酸酯模拟物的核苷和寡核苷酸。 本发明的一个方面涉及包含式(Ia)的二核苷酸的修饰核苷和寡核苷酸。 本发明的另一方面涉及一种抑制基因表达的方法,所述方法包括(a)使本发明的寡核苷酸与细胞接触; 和(b)将来自步骤(a)的细胞维持足以获得靶基因的mRNA降解的时间。
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40.
公开(公告)号:US20120095075A1
公开(公告)日:2012-04-19
申请号:US13211150
申请日:2011-08-16
申请人: Muthiah MANOHARAN , Kallanthottathil G. Rajeev , David Butler , Narayanannair K. Jayaprakash , Muthusamy Jayaraman , Laxman Eltepu
发明人: Muthiah MANOHARAN , Kallanthottathil G. Rajeev , David Butler , Narayanannair K. Jayaprakash , Muthusamy Jayaraman , Laxman Eltepu
IPC分类号: A61K31/7088 , C07D491/113 , A61K31/713 , C07D491/18 , C07H15/26 , A61K47/44 , C07D491/056 , C07D491/153
摘要: The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells. In particular, the invention provides lipids having the following structure:
摘要翻译: 本发明提供有利地用于脂质颗粒以用于体内递送治疗剂至细胞的脂质。 特别地,本发明提供具有以下结构的脂质:
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