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11. 发明申请

WO2009142822A9 2-F MODIFIED RNA INTERFERENCE AGENTS 审中-公开
标题翻译： 2-F修饰的RNA干扰剂
公开(公告)号：WO2009142822A9
公开(公告)日：2010-01-14
申请号：PCT/US2009038433
申请日：2009-03-26
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G
发明人： MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G
IPC分类号： A61K48/00 , C12N15/11
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： This invention relates to a method of modulating the expression of a target gene in an organism comprising administering an iRNA agent, wherein the iRNA comprises at least one 2'-deoxy-2'-fluoro (2'-F) nucleotide in the antisense strand and at least one modified nucleotide in the sense strand. The invention also relates to compositions comprising a single-stranded oligonucleotide that contains at least one 2'-deoxy-2'-fluoro (2'-F) nucleotide. siRNA molecule containing these oligonucleotides have decreased immunogenicity.
摘要翻译：本发明涉及一种调节生物体内靶基因表达的方法，包括给予iRNA试剂，其中iRNA在反义链中包含至少一个2'-脱氧-2'-氟（2'-F）核苷酸和有义链中至少一个修饰的核苷酸。本发明还涉及包含含有至少一个2'-脱氧-2'-氟（2'-F）核苷酸的单链寡核苷酸的组合物。含有这些寡核苷酸的siRNA分子具有降低的免疫原性。

12. 发明申请

WO2011133876A3 OLIGONUCLEOTIDES COMPRISING ACYCLIC AND ABASIC NUCLEOSIDES AND ANALOGS 审中-公开
标题翻译：包含ACYCLIC和ABASIC NUCLEOSIDES和模拟物的寡核苷酸
公开(公告)号：WO2011133876A3
公开(公告)日：2013-05-16
申请号：PCT/US2011033598
申请日：2011-04-22
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , LACKEY JEREMY , JAYAPRAKASH NARAYANANNAIR K
发明人： MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , LACKEY JEREMY , JAYAPRAKASH NARAYANANNAIR K
IPC分类号： C07H21/00
CPC分类号： C07H21/02 , C07F9/6512 , C07F9/6552 , C07F9/65583 , C07H19/20 , C07H21/00 , C12N15/111 , C12N2310/14 , C12N2310/32 , C12N2310/332
摘要： This invention relates to acyclic and abasic nucleosides and oligonucleotides prepared therefrom. For instance, oligonucleotides can be prepared having one or more of the following formulas (Iota-IIIota):, or isomers thereof.
摘要翻译：本发明涉及无环和非碱性核苷及其制备的寡核苷酸。例如，可以制备具有一个或多个下式（Iota-IIIota）：或其异构体的寡核苷酸。

13. 发明申请

WO2010039548A3 CHEMICAL MODIFICATIONS OF MONOMERS AND OLIGONUCLEOTIDES WITH CYCLOADDITION 审中-公开
标题翻译：单体和二聚体的化学修饰与循环
公开(公告)号：WO2010039548A3
公开(公告)日：2012-10-04
申请号：PCT/US2009058084
申请日：2009-09-23
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , YAMADA TAKESHI , BUTLER DAVID , JAYAPRAKASH K NARAYANANNAIR , JAYRAMAN MUTHUSAMY , MATSUDA SHIGEO , PANDEY RAJENDRA K , PENG CHANG GENG
发明人： MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , YAMADA TAKESHI , BUTLER DAVID , JAYAPRAKASH K NARAYANANNAIR , JAYRAMAN MUTHUSAMY , MATSUDA SHIGEO , PANDEY RAJENDRA K , PENG CHANG GENG
IPC分类号： C07H21/00 , A61K31/7088 , A61P31/12
CPC分类号： C12N15/113 , A61K31/713 , A61K47/544 , A61K47/64 , C07H21/00 , C12N2310/14 , C12N2310/141 , C12N2310/3513 , C12N2310/3515 , H04L65/602 , H04L67/36
摘要： The invention features compounds of formula (I) or (II). In one embodiment, the invention relates compounds and processes for conjugating ligand to oligonucleotide. The invention further relates to methods for treating various disorders and diseases such as viral infections, bacterial infections, parasitic infections, cancers, allergies, autoimmune diseases, immunodeficiencies and immunosuppression.
摘要翻译：本发明具有式（I）或（II）化合物。在一个实施方案中，本发明涉及将配体与寡核苷酸缀合的化合物和方法。本发明还涉及治疗各种病症和疾病如病毒感染，细菌感染，寄生虫感染，癌症，过敏症，自身免疫疾病，免疫缺陷和免疫抑制的方法。

14. 发明申请

WO2009082607A8 TARGETING LIPIDS 审中-公开
标题翻译：目标人物
公开(公告)号：WO2009082607A8
公开(公告)日：2010-01-28
申请号：PCT/US2008085582
申请日：2008-12-04
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , NARAYANANNAIR JAYAPRAKASH K , JAYARAMAN MUTHUSAMY
发明人： MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , NARAYANANNAIR JAYAPRAKASH K , JAYARAMAN MUTHUSAMY
IPC分类号： A61K47/48
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 , A61K48/00 , C07H21/02 , Y02P20/55
摘要： The present invention provides targeting lipids of structure (Cl) L100 - linker - L101, 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 aicd to cells in vivo.
摘要翻译：本发明提供结构（C1）L100-接头-L101的靶向脂质，其中L100是脂质，亲脂性，烷基，烯基或炔基，L101是配体或-CH 2 CH 2（OCH 2 CH 2）p O（CH 2）q CH 2 - 配体，p 为1-1000，q为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，并将包封的核酸在体内有效递送到细胞。

15. 发明申请

WO2009120878A2 NON-NATURAL RIBONUCLEOTIDES, AND METHODS OF USE THEREOF 审中-公开
标题翻译：非天然核糖核苷酸，及其使用方法
公开(公告)号：WO2009120878A2
公开(公告)日：2009-10-01
申请号：PCT/US2009038425
申请日：2009-03-26
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G
发明人： MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G
IPC分类号： C07H19/073 , C07H19/173 , C07H19/23 , C07H21/04 , C12N15/11
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包含这种修饰的核苷的修饰的核苷和寡核苷酸。本发明的另一个方面涉及一种抑制呼叫中基因表达的方法，该方法包括：（a）使本发明的寡核苷酸与细胞接触; 和（b）将来自步骤（a）的细胞维持足够的时间以获得靶基因的mRNA的降解。

16. 发明申请

WO2009100351A2 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：WO2009100351A2
公开(公告)日：2009-08-13
申请号：PCT/US2009033420
申请日：2009-02-06
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , SAH DINAH , QUERBES WILLIAM , TAN PAMELA , CHEN QINGMIN
发明人： MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , SAH DINAH , QUERBES WILLIAM , TAN PAMELA , CHEN QINGMIN
IPC分类号： A61K48/00 , C12N15/11 , C12N15/113
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： This invention relates to methods of delivering an siRNA to the central nervous system of a subject by localized delivery to oligodendrocytes.
摘要翻译：本发明涉及通过局部递送到少突胶质细胞将siRNA递送至受试者的中枢神经系统的方法。

17. 发明申请

WO2007021896A3 CHEMICALLY MODIFIED OLIGONUCLEOTIDES FOR USE IN MODULATING MICRO RNA AND USES THEREOF 审中-公开
标题翻译：用于调节微RNA的化学修饰的寡核苷酸及其用途
公开(公告)号：WO2007021896A3
公开(公告)日：2009-06-11
申请号：PCT/US2006031313
申请日：2006-08-10
申请人： ALNYLAM PHARMACEUTICALS INC , UNIV ROCKEFELLER , STOFFEL MARKUS , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G
发明人： STOFFEL MARKUS , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G
IPC分类号： C07H21/00 , A61K48/00
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/345 , C12N2310/346 , C12N2310/3515 , C12N2310/3521 , C12N2310/3527 , C12N2310/3531 , C12N2310/3533
摘要： This invention relates generally to chemically modified oligonuceotides useful for modulating expression of microRNAs and pre-microRNAs. More particularly, the invention relates to single stranded chemically modified oligonuceotides for inhibiting microRNA and pre-microRNA expression 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表达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表达的单链化学修饰的寡核苷酸以及制备和使用修饰的寡核苷酸的方法。

18. 发明申请

WO2006112872A3 OLIGONUCLEOTIDES COMPRISING A LIGAND TETHERED TO A MODIFIED OR NON-NATURAL NUCLEOBASE 审中-公开
标题翻译：包含配对或修饰的非天然核苷酸的配体的寡核苷酸
公开(公告)号：WO2006112872A3
公开(公告)日：2008-07-17
申请号：PCT/US2005027722
申请日：2005-08-04
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , XIA JIE
发明人： MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , XIA JIE
IPC分类号： C07H21/00 , A61K31/7115
CPC分类号： C07H21/00 , A61K31/7115 , C07F9/6561 , C07H21/02 , C12Q1/6883
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 robose 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，天然存在于核苷中的罗糖糖部分被己糖，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。

19. 发明申请

WO2006093526A3 OLIGONUCLEOTIDES COMPRISING A MODIFIED OR NON-NATURAL NUCLEOBASE 审中-公开
标题翻译：包含改性或非天然核苷酸的寡核苷酸
公开(公告)号：WO2006093526A3
公开(公告)日：2008-01-24
申请号：PCT/US2005025967
申请日：2005-07-21
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , XIA JIE , RAJEEV KALLANTHOTTATHIL G
发明人： MANOHARAN MUTHIAH , XIA JIE , RAJEEV KALLANTHOTTATHIL G
IPC分类号： C07H21/00 , A61K31/7115
CPC分类号： C07H21/00 , A61K31/7115 , C07F9/65515 , C07F9/65586 , C07F9/6561 , C07H21/02
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 e4mbodiments, 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 phosphrothioate 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磷酸硫酸酯键取代。

20. 发明申请

WO2005115481A2 NUCLEASE RESISTANT DOUBLE-STRANDED RIBONUCLEIC ACID 审中-公开
标题翻译：抗核抗原双重抗坏血酸
公开(公告)号：WO2005115481A2
公开(公告)日：2005-12-08
申请号：PCT/US2005018931
申请日：2005-05-27
申请人： ALNYLAM PHARMACEUTICALS INC , VORNLOCHER HANS-PETER , HADWIGER PHILIPP , ZIMMERMANN TRACY STAGE , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , ROEHL INGO , AKINC AKIN
发明人： VORNLOCHER HANS-PETER , HADWIGER PHILIPP , ZIMMERMANN TRACY STAGE , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , ROEHL INGO , AKINC AKIN
IPC分类号： A61K48/00 , C12N15/113
CPC分类号： C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/3231 , C12N2310/53 , C12N2320/51 , C12N2310/3521 , C12N2310/3525 , C12N2310/3527
摘要： This invention relates to modified double-stranded oligoribonucleic acid (dsRNA) having improved stability in cells and biological fluids, and methods of making and identifying dsRNA having improved stability, and of using the dsRNA to inhibit the expression or function of a target gene.
摘要翻译：本发明涉及在细胞和生物流体中具有改善的稳定性的修饰的双链寡核糖核酸（dsRNA），以及制备和鉴定具有改善的稳定性的dsRNA的方法，以及使用dsRNA抑制靶基因的表达或功能。

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