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41. 发明申请

US20130018085A1 iRNA Agents Targeting VEGF 审中-公开
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.

42. 发明授权

US08344125B2 Modified iRNA agents 有权
标题翻译：修饰的iRNA试剂
公开(公告)号：US08344125B2
公开(公告)日：2013-01-01
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： C07H21/04 , A61K31/70
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摘要： The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose.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。

43. 发明申请

US20120095075A1 NOVEL LIPIDS AND COMPOSITIONS FOR THE DELIVERY OF THERAPEUTICS 审中-公开
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：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：

44. 发明申请

US20120046478A1 LIPID CONTAINING FORMULATIONS 有权
标题翻译：含脂制剂
公开(公告)号：US20120046478A1
公开(公告)日：2012-02-23
申请号：US13211094
申请日：2011-08-16
申请人： Muthiah MANOHARAN , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
发明人： Muthiah MANOHARAN , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
IPC分类号： C07C271/16 , C07C235/74 , C07C69/40
CPC分类号： C07C219/06 , A61K9/1272 , A61K9/1273 , A61K31/325 , A61K47/34 , C07C209/78 , C07C233/36 , C07C271/10 , C07C323/25
摘要： Compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes are described.
摘要翻译：描述了用于施用基于核酸的疗法，例如缔合复合物如脂质体和脂质复合物的组合物和方法。

45. 发明申请

US20120027803A1 BIODEGRADABLE LIPIDS FOR THE DELIVERY OF ACTIVE AGENTS 有权
标题翻译：用于交付活性剂的生物可降解脂肪
公开(公告)号：US20120027803A1
公开(公告)日：2012-02-02
申请号：US13153200
申请日：2011-06-03
申请人： Muthiah MANOHARAN , Martin Maier , Muthusamy Jayaraman , Shigeo Matsuda , Narayanannair K. Jayaprakash , Kallanthottathil G. Rajeev , Akin Akinc , Thomas A. Baillie
发明人： Muthiah MANOHARAN , Martin Maier , Muthusamy Jayaraman , Shigeo Matsuda , Narayanannair K. Jayaprakash , Kallanthottathil G. Rajeev , Akin Akinc , Thomas A. Baillie
IPC分类号： A61K47/44 , A61K31/713 , A61P37/04 , C12N5/071 , C12N15/85 , C07C229/30 , A61K31/7088
CPC分类号： C07C229/12 , A61K31/713 , A61K39/0005 , A61K47/44 , C12N15/113 , C12N15/88 , C12N2310/14 , C12N2320/30
摘要： The present invention relates to a cationic lipid having one or more biodegradable groups located in the mid- or distal section of 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.
摘要翻译：本发明涉及一种阳离子脂质，其具有位于阳离子脂质的脂质部分（例如，疏水链）的中间或远端部分中的一个或多个可生物降解基团。这些阳离子脂质可以掺入用于递送活性剂（例如核酸）的脂质颗粒中。本发明还涉及包含中性脂质，能够减少聚集的脂质，本发明的阳离子脂质和任选的固醇的脂质颗粒。脂质颗粒可以进一步包括治疗剂如核酸。

46. 发明授权

US08058448B2 Processes and reagents for sulfurization of oligonucleotides 有权
标题翻译：寡核苷酸硫化的方法和试剂
公开(公告)号：US08058448B2
公开(公告)日：2011-11-15
申请号：US12351605
申请日：2009-01-09
申请人： Muthiah Manoharan , Michael E. Jung , Kallanthottathil G. Rajeev , Rajendra K. Pandey , Gang Wang
发明人： Muthiah Manoharan , Michael E. Jung , Kallanthottathil G. Rajeev , Rajendra K. Pandey , Gang Wang
IPC分类号： C07D285/01 , C07H21/00
CPC分类号： C07H21/00 , C07H21/04 , Y02P20/55
摘要： The present invention relates to processes and reagents for oligonucleotide synthesis and purification. One aspect of the present invention relates to compounds useful for activating phosphoramidites in oligonucleotide synthesis. Another aspect of the present invention relates to a method of preparing oligonucleotides via the phosphoramidite method using an activator of the invention. Another aspect of the present invention relates to sulfur-transfer agents. In a preferred embodiment, the sulfur-transfer agent is a 3-amino-1,2,4-dithiazolidine-5-one. Another aspect of the present invention relates to a method of preparing a phosphorothioate by treating a phosphite with a sulfur-transfer reagent of the invention. In a preferred embodiment, the sulfur-transfer agent is a 3-amino-1,2,4-dithiazolidine-5-one. Another aspect of the present invention relates to compounds that scavenge acrylonitrile produced during the deprotection of phosphate groups bearing ethylnitrile protecting groups. In a preferred embodiment, the acrylonitrile scavenger is a polymer-bound thiol. Another aspect of the present invention relates to agents used to oxidize a phosphite to a phosphate. In a preferred embodiment, the oxidizing agent is sodium chlorite, chloramine, or pyridine-N-oxide. Another aspect of the present invention relates to methods of purifying an oligonucleotide by annealing a first single-stranded oligonucleotide and second single-stranded oligonucleotide to form a double-stranded oligonucleotide; and subjecting the double-stranded oligonucleotide to chromatographic purification. In a preferred embodiment, the chromatographic purification is high-performance liquid chromatography.
摘要翻译：本发明涉及寡核苷酸合成和纯化的方法和试剂。本发明的一个方面涉及可用于在寡核苷酸合成中活化亚磷酰胺的化合物。本发明的另一方面涉及使用本发明的活化剂通过亚磷酰胺方法制备寡核苷酸的方法。本发明的另一方面涉及硫转移剂。在优选的实施方案中，硫转移剂是3-氨基-1,2,4-二噻唑烷-5-酮。本发明的另一方面涉及通过用本发明的硫转移试剂处理亚磷酸酯制备硫代磷酸酯的方法。在优选的实施方案中，硫转移剂是3-氨基-1,2,4-二噻唑烷-5-酮。本发明的另一方面涉及清除在带有乙腈基保护基团的磷酸酯基团去保护过程中产生的丙烯腈的化合物。在优选的实施方案中，丙烯腈清除剂是聚合物结合的硫醇。本发明的另一方面涉及用于将亚磷酸盐氧化成磷酸盐的试剂。在优选的实施方案中，氧化剂是亚氯酸钠，氯胺或吡啶-N-氧化物。本发明的另一方面涉及通过退火第一单链寡核苷酸和第二单链寡核苷酸来纯化寡核苷酸以形成双链寡核苷酸的方法; 并对双链寡核苷酸进行色谱纯化。在优选的实施方案中，色谱纯化是高效液相色谱法。

47. 发明授权

US08017763B2 Chemically modified oligonucleotides 有权
标题翻译：化学修饰的寡核苷酸
公开(公告)号：US08017763B2
公开(公告)日：2011-09-13
申请号：US12561529
申请日：2009-09-17
申请人： Muthiah Manoharan , Venkitasamy Kesavan , Kallanthottathil G. Rajeev
发明人： Muthiah Manoharan , Venkitasamy Kesavan , Kallanthottathil G. Rajeev
IPC分类号： C07H21/04 , A61K31/70
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.
摘要翻译：本发明涉及用于制备和使用化学修饰的寡核苷酸抑制基因表达的组合物和方法。

48. 发明授权

US07947659B2 iRNA agents targeting VEGF 有权
标题翻译：靶向VEGF的iRNA试剂
公开(公告)号：US07947659B2
公开(公告)日：2011-05-24
申请号：US11078073
申请日：2005-03-11
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： A61K31/70 , C07H21/04
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.
摘要翻译：本发明的特征涉及用于调节血管内皮生长因子（VEGF）表达的化合物，组合物和方法，例如通过RNA干扰（RNAi）机制。化合物和组合物包括可以未修饰或化学修饰的iRNA试剂。

49. 发明申请

US20110118340A1 DELIVERY OF RNAI CONSTRUCTS TO OLIGODENDROCYTES 审中-公开
标题翻译：将RNAI结构递送到寡糖脱乙酰壳多糖
公开(公告)号：US20110118340A1
公开(公告)日：2011-05-19
申请号：US12866444
申请日：2009-02-06
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Dinah Sah , William Querbes , Pamela Tan , Qingmin Chen
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Dinah Sah , William Querbes , Pamela Tan , Qingmin Chen
IPC分类号： A61K31/7088 , A61P31/12 , A61P25/00 , A61P25/18 , A61P25/16 , A61P25/28
CPC分类号： C12N15/1131 , A61K47/551 , A61K47/554 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2320/11 , C12N2320/32 , C12N2310/3521
摘要： The invention provides methods for delivering a double-stranded nbonucleic acid (dsRNA) to the central nervous system of a subject, and particularly, to oligodendrocytes of a subject by localized delivery to the brain, e.g., to the corpus caïlosum. For example, the dsRNA molecules can include a first sequence that is selected from the Sroup consisting of the sense sequences of Tables 8, 10, 13-16, and a second sequence selected from the group consisting of the antisense sequences of Tables 8, 10, and 13-16. The dsRNA molecules can include naturally occurring nucleotides or can include at least one modified nucleotide, such as a 2′-O-methyl modified nucleotide, a nucleotide comprising a 5′-phosphorothioate group, or a terminal nucleotide linked to a conjugate group, such as to a cholesteryl derivative or a vitamin E group. Alternatively, the modified nucleotide may be chosen from the group consisting of a 2f-deoxy-2′-fliιioro modified nucleotide, a 2′-de-oxy-modified nucleotide, a locked nucleotide, an abasic nucleotide, 2′-amino-modified nucleotide, 2′-alkyl-modified nucleotide, morpholino nucleotide, a phosphoramidate, and a non-natural bas comprising nucleotide. Generally, such modified sequences will be based on a first sequence of a dsRNA selected from the group consisting of the sense sequences of Tables 8, 10, and 13-16, and a second sequence selected from the group consisting of the antisense sequences of Tables 8 10, and 13-16.
摘要翻译：本发明提供了将双链核糖核酸（dsRNA）递送到受试者的中枢神经系统，特别是通过局部递送至脑的例如对胼the体的受试者的少突胶质细胞的方法。例如，dsRNA分子可以包括选自由表8,10,13-16的有义序列组成的组的第一序列和选自表8,10的反义序列的第二序列，和13-16。 dsRNA分子可以包括天然存在的核苷酸，或者可以包括至少一个修饰的核苷酸，例如2'-O-甲基修饰的核苷酸，包含5'-硫代磷酸酯基团的核苷酸或与缀合物连接的末端核苷酸，关于胆固醇衍生物或维生素E组。或者，修饰的核苷酸可以选自2f-脱氧-2'-氟修饰的核苷酸，2'-脱氧修饰的核苷酸，锁定的核苷酸，无碱基的核苷酸，2'-氨基修饰的核苷酸，2'-烷基修饰的核苷酸，吗啉代核苷酸，氨基磷酸酯和包含核苷酸的非天然碱基。通常，这样的修饰序列将基于选自表8,10和13-16的有义序列的dsRNA的第一序列和选自下表的反义序列的第二序列 8 10和13-16。

50. 发明授权

US07632932B2 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：US07632932B2
公开(公告)日：2009-12-15
申请号：US11197753
申请日：2005-08-04
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Jie Xia
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Jie Xia
IPC分类号： C07H21/00 , C07H21/02 , 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。

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