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61. 发明申请

US20120027796A1 NOVEL LIPIDS AND COMPOSITIONS FOR THE DELIVERY OF THERAPEUTICS 有权
标题翻译：用于输送治疗药物的新型脂肪和组合物
公开(公告)号：US20120027796A1
公开(公告)日：2012-02-02
申请号：US13128253
申请日：2009-11-10
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev , David Butler , Jayaprakash K. Narayanannair , Muthusamy Jayaraman , Laxmab Eltepu
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , David Butler , Jayaprakash K. Narayanannair , Muthusamy Jayaraman , Laxmab Eltepu
IPC分类号： A61K39/12 , C07D317/44 , C07D317/46 , C07D493/04 , C07D317/70 , C07D491/113 , C07D317/28 , C07D317/72 , C07F9/655 , A61K47/44 , A61K31/7088 , A61K31/713 , C12N5/071 , A61K39/39 , A61K39/00 , A61K39/02 , A61P37/04 , A61P31/04 , A61P31/12 , A61P33/00 , A61P35/00 , C07D491/056
CPC分类号： A61K47/44 , A61K9/1272 , A61K31/7088 , A61K31/713 , A61K39/00 , A61K39/39 , A61K47/10 , A61K47/18 , A61K47/20 , A61K47/28 , A61K2039/55555 , A61K2039/55561 , C07C229/08 , C07C229/30 , C07C237/16 , C07C251/38 , C07C251/78 , C07C271/12 , C07C271/20 , C07C323/25 , C07D203/10 , C07D317/28 , C07D317/44 , C07D317/46 , C07D317/72 , C07D319/06 , C07D405/12 , C07D491/056 , C07D491/113 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/3515 , C12N2320/32 , Y02A50/464
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 structures: (Formula (I) or (XXXV)).
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：（式（I）或（XXXV））。

62. 发明申请

US20110257244A1 CHEMICALLY MODIFIED OLIGONUCLEOTIDES AND SMALL MOLECULES FOR USE IN REDUCING MICRO RNA ACTIVITY LEVELS AND USES THEREOF 审中-公开
标题翻译：化学修饰的寡核苷酸和用于减少微RNA活性水平的小分子及其用途
公开(公告)号：US20110257244A1
公开(公告)日：2011-10-20
申请号：US12863369
申请日：2009-01-16
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev
IPC分类号： A61K31/713 , A61P35/00 , A61P19/06 , A61P7/06 , A61P25/28
CPC分类号： C12N15/113 , C12N2310/113 , C12N2310/14 , C12N2310/33 , C12N2310/351
摘要： This invention relates generally to chemically modified oligonucleotides useful for modulating activity of microRNAs and pre-microRNAs. More particularly, the invention relates to single stranded chemically modified oligonucleotides for inhibiting microRNA and pre-microRNA activity and to methods of making and using the modified oligonucleotides.
摘要翻译：本发明一般涉及可用于调节微小RNA和pre-microRNAs的活性的化学修饰的寡核苷酸。更具体地说，本发明涉及用于抑制微小RNA和pre-microRNA活性的单链化学修饰的寡核苷酸以及制备和使用经修饰的寡核苷酸的方法。

63. 发明申请

US20110130440A1 NON-NATURAL RIBONUCLEOTIDES, AND METHODS OF USE THEREOF 审中-公开
标题翻译：非天然抗肿瘤药物及其使用方法
公开(公告)号：US20110130440A1
公开(公告)日：2011-06-02
申请号：US12934323
申请日：2009-03-26
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev
IPC分类号： A61K31/7105 , C07H19/23 , C07H7/06 , C07H19/06 , C07H21/00 , C07H21/02 , C12N5/071
CPC分类号： C07H19/073 , C07H19/173 , C07H19/23 , C07H21/04 , C12N15/111 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2320/51 , C12N2310/3521 , C12N2310/3525
摘要： One aspect of the present invention relates to modified nucleosides and oligonucleotides comprising such modified nucleosides. 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：本发明的一个方面涉及修饰的核苷和包含这种修饰的核苷的寡核苷酸。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。

64. 发明授权

US07893224B2 Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase 有权
标题翻译：包含与修饰或非天然核碱基连接的配体的寡核苷酸
公开(公告)号：US07893224B2
公开(公告)日：2011-02-22
申请号：US12533710
申请日：2009-07-31
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Jie Xia
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Jie Xia
IPC分类号： C07G3/00 , C07H21/04 , C12Q1/68
CPC分类号： C07H21/00 , A61K31/7115 , C07F9/6561 , C07H21/02 , C12Q1/6883 , Y02A50/385 , Y02A50/387 , Y02A50/393 , Y02A50/395 , Y02A50/411 , Y02A50/463 , Y02A50/465 , Y02A50/467
摘要： One aspect of the present invention relates to a double-stranded oligonucleotide comprising at least one ligand tethered to an altered or non-natural nucleobase. In certain embodiments, the non-natural nucleobase is difluorotolyl, nitropyrrolyl, or nitroimidazolyl. In certain embodiments, the ligand is a steroid or aromatic compound. In certain embodiments, only one of the two oligonucleotide strands comprising the double-stranded oligonucleotide contains a ligand tethered to an altered or non-natural nucleobase. In certain embodiments, both of the oligonucleotide strands comprising the double-stranded oligonucleotide independently contain a ligand tethered to an altered or non-natural nucleobase. In certain embodiments, the oligonucleotide strands comprise at least one modified sugar moiety. Another aspect of the present invention relates to a single-stranded oligonucleotide comprising at least one ligand tethered to an altered or non-natural nucleobase. In certain embodiments, the non-natural nucleobase is difluorotolyl, nitropyrrolyl, or nitroimidazolyl. In certain embodiments, the ligand is a steroid or aromatic compound. In certain embodiments, the ribose sugar moiety that occurs naturally in nucleosides is replaced with a hexose sugar, polycyclic heteroalkyl ring, or cyclohexenyl group. In certain embodiments, at least one phosphate linkage in the oligonucleotide has been replaced with a phosphorothioate linkage.
摘要翻译：本发明的一个方面涉及包含至少一个与改变的或非天然核碱基连接的配体的双链寡核苷酸。在某些实施方案中，非天然核碱基是二氟苯甲酰基，硝基吡咯基或硝基咪唑基。在某些实施方案中，配体是类固醇或芳族化合物。在某些实施方案中，包含双链寡核苷酸的两条寡核苷酸链中只有一条含有与改变的或非天然核碱基连接的配体。在某些实施方案中，包含双链寡核苷酸的两条寡核苷酸链都独立地包含与改变的或非天然核碱基连接的配体。在某些实施方案中，寡核苷酸链包含至少一个修饰的糖部分。本发明的另一方面涉及包含至少一个与改变的或非天然核碱基连接的配体的单链寡核苷酸。在某些实施方案中，非天然核碱基是二氟苯甲酰基，硝基吡咯基或硝基咪唑基。在某些实施方案中，配体是类固醇或芳族化合物。在某些实施方案中，在核苷中天然存在的核糖部分被己糖，多环杂烷基环或环己烯基取代。在某些实施方案中，寡核苷酸中的至少一个磷酸键被硫代磷酸酯键取代。

65. 发明申请

US20100324120A1 LIPID FORMULATION 有权
标题翻译：脂质配方
公开(公告)号：US20100324120A1
公开(公告)日：2010-12-23
申请号：US12813448
申请日：2010-06-10
申请人： Jianxin Chen , Steven Ansell , Akin Akinc , Joseph Robert Dorkin , Xiaojun Qin , William Cantley , Muthiah Manoharan , Kallanthottathil G. Rajeev , Jayaprakash K. Narayanannair , Muthusamy Jayaraman
发明人： Jianxin Chen , Steven Ansell , Akin Akinc , Joseph Robert Dorkin , Xiaojun Qin , William Cantley , Muthiah Manoharan , Kallanthottathil G. Rajeev , Jayaprakash K. Narayanannair , Muthusamy Jayaraman
IPC分类号： A61K31/711 , C07C229/30 , A61K47/16 , A61K31/713 , A61K47/26 , C12N5/00 , A61P43/00
CPC分类号： A61K9/1272 , A61K9/1278 , A61K31/22 , A61K31/7088 , A61K31/712 , A61K31/713 , A61K38/1709 , A61K38/4846 , A61K47/14 , A61K47/549 , A61K47/551 , A61K47/6911 , A61K48/0033 , C07C29/58 , C07C227/18 , C07C227/40 , C07C229/12 , C07D475/04 , C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/32 , Y02A50/463 , A61K2300/00 , C12N2310/3521 , C12N2310/3533
摘要： The invention features a cationic lipid of formula I, an improved lipid formulation comprising a cationic lipid of formula I and corresponding methods of use. Also disclosed are targeting lipids, and specific lipid formulations comprising such targeting lipids.
摘要翻译：本发明的特征在于式I的阳离子脂质，包含式I的阳离子脂质和相应的使用方法的改进的脂质制剂。还公开了靶向脂质和包含这种靶向脂质的特定脂质制剂。

66. 发明授权

US07772387B2 Oligonucleotides comprising a modified or non-natural nucleobase 有权
标题翻译：包含修饰或非天然核碱基的寡核苷酸
公开(公告)号：US07772387B2
公开(公告)日：2010-08-10
申请号：US12495932
申请日：2009-07-01
申请人： Muthiah Manoharan , Jie Xia , Kallanthottathil G. Rajeev
发明人： Muthiah Manoharan , Jie Xia , Kallanthottathil G. Rajeev
IPC分类号： C07H21/02 , C07H21/04 , C12Q1/68
CPC分类号： C07H21/00 , A61K31/7115 , C07F9/65515 , C07F9/65586 , C07F9/6561 , C07H21/02 , Y02A50/385 , Y02A50/387 , Y02A50/393 , Y02A50/395 , Y02A50/411 , Y02A50/463 , Y02A50/465 , Y02A50/467
摘要： One aspect of the present invention relates to a double-stranded oligonucleotide comprising at least one non-natural nucleobase. In certain embodiments, the non-natural nucleobase is difluorotolyl, nitroindolyl, nitropyrrolyl, or nitroimidazolyl. In a preferred embodiment, the non-natural nucleobase is difluorotolyl. In certain embodiments, only one of the two oligonucleotide strands comprising the double-stranded oligonucleotide contains a non-natural nucleobase. In certain embodiments, both of the oligonucleotide strands comprising the double-stranded oligonucleotide independently contain a non-natural nucleobase. In certain embodiments, the oligonucleotide strands comprise at least one modified sugar moiety. Another aspect of the present invention relates to a single-stranded oligonucleotide comprising at least one non-natural nucleobase. In a preferred embodiment, the non-natural nucleobase is difluorotolyl. In certain embodiments, the ribose sugar moiety that occurs naturally in nucleosides is replaced with a hexose sugar, polycyclic heteroalkyl ring, or cyclohexenyl group. In certain embodiments, at least one phosphate linkage in the oligonucleotide has been replaced with a phosphorothioate linkage.
摘要翻译：本发明的一个方面涉及包含至少一种非天然核碱基的双链寡核苷酸。在某些实施方案中，非天然核碱基是二氟苯甲酰基，硝基吲哚基，硝基吡咯基或硝基咪唑基。在优选的实施方案中，非天然核碱基是二氟苯甲酰基。在某些实施方案中，包含双链寡核苷酸的两条寡核苷酸链中只有一条含有非天然核碱基。在某些实施方案中，包含双链寡核苷酸的两条寡核苷酸链都独立地含有非天然核碱基。在某些实施方案中，寡核苷酸链包含至少一个修饰的糖部分。本发明的另一方面涉及包含至少一种非天然核碱基的单链寡核苷酸。在优选的实施方案中，非天然核碱基是二氟苯甲酰基。在某些实施方案中，在核苷中天然存在的核糖部分被己糖，多环杂烷基环或环己烯基取代。在某些实施方案中，寡核苷酸中的至少一个磷酸键被硫代磷酸酯键取代。

67. 发明申请

US20100179309A1 MODIFIED iRNA AGENTS 有权
标题翻译：改性iRNA代理
公开(公告)号：US20100179309A1
公开(公告)日：2010-07-15
申请号：US12619382
申请日：2009-11-16
申请人： Muthiah MANOHARAN , Kallanthottathil G. Rajeev
发明人： Muthiah MANOHARAN , Kallanthottathil G. Rajeev
IPC分类号： C07H21/02
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。

68. 发明授权

US07723509B2 IRNA agents with biocleavable tethers 有权
标题翻译：具有生物可清除系绳的IRNA试剂
公开(公告)号：US07723509B2
公开(公告)日：2010-05-25
申请号：US10985426
申请日：2004-11-09
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev
IPC分类号： C07H21/04 , A61K31/70
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/14 , C12N2310/323 , C12N2310/3511 , 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 that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent.
摘要翻译：本发明涉及iRNA试剂，其优选包括其中核糖部分已被核糖替代的单体，其进一步包括具有一个或多个连接基团的系链，其中至少一个连接基团是可切割的连接组。系链又可以连接到所选择的部分，例如配体，例如靶向或递送部分，或改变物理性质的部分。可切割连接基团是在细胞外部足够稳定的连接基团，使得其允许靶向治疗有益量的iRNA试剂（例如，单链或双链iRNA试剂），其通过可切割连接基团连接至靶向细胞靶向细胞，但是在进入靶细胞时被切割以从靶向剂释放iRNA试剂。

69. 发明申请

US20090247608A1 Targeting Lipids 审中-公开
标题翻译：靶向脂质
公开(公告)号：US20090247608A1
公开(公告)日：2009-10-01
申请号：US12328669
申请日：2008-12-04
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Gayaprakash K. Narayananovair , Muthusamy Jayaraman
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Gayaprakash K. Narayananovair , Muthusamy Jayaraman
IPC分类号： A61K31/713 , C07K14/00 , A61K47/42 , A61K31/7088 , C07K7/04 , C07K5/04 , A61K47/12
摘要： The present invention provides targeting lipids of structure L100—linker—L101 (CI), where L100 is a lipid, lipophile, alkyl, alkenyl or alkynyl, L101 is a ligand or —CH2CH2(OCH2CH2)pO(CH2)qCH2-ligand, p is 1-1000, and q is 1-20. In addition, the invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo.
摘要翻译：本发明提供了结构<？in-line-formula description =“In-line Formulas”end =“lead”？> L100-linker-L101（CI），<？in-line-formula description =“In 其中L100是脂质，亲脂性，烷基，烯基或炔基，L101是配体或-CH2CH2（OCH2CH2）pO（CH2）qCH2-配体，p是1-1000，和 q是1-20。此外，本发明提供了将治疗剂递送至细胞的组合物和方法。特别地，这些包括提供核酸的有效包封的新型脂质和核酸 - 脂质颗粒，并将包封的核酸有效地递送到细胞体内。

70. 发明授权

US07579451B2 Oligonucleotides comprising a modified or non-natural nucleobase 有权
标题翻译：包含修饰或非天然核碱基的寡核苷酸
公开(公告)号：US07579451B2
公开(公告)日：2009-08-25
申请号：US11186915
申请日：2005-07-21
申请人： Muthiah Manoharan , Jie Xia , Kallanthottathil G. Rajeev
发明人： Muthiah Manoharan , Jie Xia , Kallanthottathil G. Rajeev
IPC分类号： C07H21/00 , C07H21/02 , C07H21/04 , C12Q1/68
CPC分类号： C07H21/00 , A61K31/7115 , C07F9/65515 , C07F9/65586 , C07F9/6561 , C07H21/02 , Y02A50/385 , Y02A50/387 , Y02A50/393 , Y02A50/395 , Y02A50/411 , Y02A50/463 , Y02A50/465 , Y02A50/467
摘要： One aspect of the present invention relates to a double-stranded oligonucleotide comprising at least one non-natural nucleobase. In certain embodiments, the non-natural nucleobase is difluorotolyl, nitroindolyl, nitropyrrolyl, or nitroimidazolyl. In a preferred embodiment, the non-natural nucleobase is difluorotolyl. In certain embodiments, only one of the two oligonucleotide strands comprising the double-stranded oligonucleotide contains a non-natural nucleobase. In certain embodiments, both of the oligonucleotide strands comprising the double-stranded oligonucleotide independently contain a non-natural nucleobase. In certain embodiments, the oligonucleotide strands comprise at least one modified sugar moiety. Another aspect of the present invention relates to a single-stranded oligonucleotide comprising at least one non-natural nucleobase. In a preferred embodiment, the non-natural nucleobase is difluorotolyl. In certain embodiments, the ribose sugar moiety that occurs naturally in nucleosides is replaced with a hexose sugar, polycyclic heteroalkyl ring, or cyclohexenyl group. In certain embodiments, at least one phosphate linkage in the oligonucleotide has been replaced with a phosphorothioate linkage.
摘要翻译：本发明的一个方面涉及包含至少一种非天然核碱基的双链寡核苷酸。在某些实施方案中，非天然核碱基是二氟苯甲酰基，硝基吲哚基，硝基吡咯基或硝基咪唑基。在优选的实施方案中，非天然核碱基是二氟苯甲酰基。在某些实施方案中，包含双链寡核苷酸的两条寡核苷酸链中只有一条含有非天然核碱基。在某些实施方案中，包含双链寡核苷酸的两条寡核苷酸链都独立地含有非天然核碱基。在某些实施方案中，寡核苷酸链包含至少一个修饰的糖部分。本发明的另一方面涉及包含至少一种非天然核碱基的单链寡核苷酸。在优选的实施方案中，非天然核碱基是二氟苯甲酰基。在某些实施方案中，在核苷中天然存在的核糖部分被己糖，多环杂烷基环或环己烯基取代。在某些实施方案中，寡核苷酸中的至少一个磷酸键被硫代磷酸酯键取代。

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