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11. 发明申请

WO2009020771A2 RNAI AGENTS COMPRISING UNIVERSAL NUCLEOBASES 审中-公开
标题翻译：包含通用核苷酸的RNAi药剂
公开(公告)号：WO2009020771A2
公开(公告)日：2009-02-12
申请号：PCT/US2008/071010
申请日：2008-07-24
申请人： ALNYLAM PHARMACEUTICALS, INC. , MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G.
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G.
IPC分类号： C12N15/09 , C07H21/00
CPC分类号： C07F9/65515 , C07F9/65586 , C07F9/6561 , C07H21/02 , C12N15/111 , C12N2310/14 , C12N2310/331 , C12N2320/34
摘要： One aspect of the present invention relates to an oligonucleotide agent comprising at least one universal nucleobase. In certain embodiments, the universal nucleobase is difluorotolyl, nitroindolyl, nitropyrrolyl, or nitroimidazolyl. In a preferred embodiment, the universal nucleobase is difluorotolyl. In certain embodiments, the oligonucleotide is double-stranded. In certain embodiments, the oligonucleotide is single-stranded. Another aspect of the present invention relates to a method of altering the expression level of a target in the presence of target sequence polymorphism. In a preferred embodiment, the oligonucleotide agent alters the expression of different alleles of a gene. In another preferred embodiment, the oligonucleotide agent alters the expression level of two or more genes. In another embodiment, the oligonucleotide agent alters the expression level of a viral gene from different strains of the virus. In another embodiment, the oligonucleotide agent alters the expression level of genes from different species.
摘要翻译：本发明的一个方面涉及包含至少一个通用核碱基的寡核苷酸试剂。在某些实施方案中，通用核碱基是二氟甲苯基，硝基吲哚基，硝基吡咯基或硝基咪唑基。在一个优选的实施方案中，通用核碱基是二氟甲苯基。在某些实施方案中，寡核苷酸是双链的。在某些实施方案中，寡核苷酸是单链的。本发明的另一方面涉及在靶序列多态性存在下改变靶标表达水平的方法。在优选的实施方案中，寡核苷酸试剂改变基因的不同等位基因的表达。在另一个优选实施方案中，寡核苷酸试剂改变两种或更多种基因的表达水平。在另一个实施方案中，寡核苷酸试剂改变来自不同病毒株的病毒基因的表达水平。在另一个实施方案中，寡核苷酸试剂改变来自不同物种的基因的表达水平。

12. 发明申请

WO2006020768A3 CHEMICALLY MODIFIED OLIGONUCLEOTIDES 审中-公开
标题翻译：化学修饰的寡核苷酸
公开(公告)号：WO2006020768A3
公开(公告)日：2007-07-05
申请号：PCT/US2005028550
申请日：2005-08-10
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , KESAVAN VENKITASAMY , RAJEEV KALLANTHOTTATHIL G
发明人： MANOHARAN MUTHIAH , KESAVAN VENKITASAMY , RAJEEV KALLANTHOTTATHIL G
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。

13. 发明申请

WO2006093526A2 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：WO2006093526A2
公开(公告)日：2006-09-08
申请号：PCT/US2005/025967
申请日：2005-07-21
申请人： ALNYLAM PHARMACEUTICALS , MANOHARAN, Muthiah , XIE, Jie , RAJEEV, Kallanthottathil, G.
发明人： MANOHARAN, Muthiah , XIE, Jie , RAJEEV, Kallanthottathil, G.
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 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。

14. 发明申请

WO2004042029A2 OLIGOMERIC COMPOUNDS HAVING MODIFIED BASES FOR BINDING TO CYTOSINE AND URACIL OR THYMINE AND THEIR USE IN GENE MODULATION 审中-公开
标题翻译：具有修饰基团的寡核苷酸化合物用于结合胞嘧啶和尿素或噻唑及其在基因调控中的用途
公开(公告)号：WO2004042029A2
公开(公告)日：2004-05-21
申请号：PCT/US2003/035146
申请日：2003-11-04
申请人： ISIS PHARMACEUTICALS, INC. , BAKER, Brenda, F. , ELDRUP, Anne, B. , MANOHARAN, Muthiah , BHAT, Balkrishen , GRIFFEY, Richard , SWAYZE, Eric, E. , CROOKE, Stanley, T. , RAJEEV, Kallanthottathil, G.
发明人： BAKER, Brenda, F. , ELDRUP, Anne, B. , MANOHARAN, Muthiah , BHAT, Balkrishen , GRIFFEY, Richard , SWAYZE, Eric, E. , CROOKE, Stanley, T. , RAJEEV, Kallanthottathil, G.
IPC分类号： C12N
CPC分类号： C07H21/00 , C07H21/04 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/343 , C12N2310/3521 , C12N2310/3533 , C12N2320/51
摘要： Oligomer compositions comprising first and second oligomers are provided wherein at least a portion Of the second oligomer, at least a portion of the first oligomer is complementary to and capble of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers has a modified base for binding to a cytosine, uracil, or thymine base in the opposite strand. Oligonucleotide&protein compositions are also provided comprising an oligomer complementartary To and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex (RISC), wherein the oligomer has a modified base for binding to a cytosine, uracil, or thymine base in the opposite strand.
摘要翻译：提供了包含第一和第二寡聚物的低聚物组合物，其中至少一部分第二寡聚体，至少一部分第一寡聚体与选定的靶核酸杂交并与其结合，并且与第一或第二寡核苷酸中的至少一个互补寡聚物具有与相对链中的胞嘧啶，尿嘧啶或胸腺嘧啶碱基结合的修饰碱基。还提供了寡核苷酸和蛋白质组合物，其包含寡核苷酸互补物，并且能够与选定的靶核酸和至少一种包含至少一部分RNA诱导的沉默复合物（RISC）的蛋白质杂交，其中所述寡聚物具有修饰的结合碱基到相反链中的胞嘧啶，尿嘧啶或胸腺嘧啶碱基。

15. 发明申请

WO2002097134A2 MODIFIED PEPTIDE NUCLEIC ACID 审中-公开
标题翻译：改性肽核酸
公开(公告)号：WO2002097134A2
公开(公告)日：2002-12-05
申请号：PCT/US2002/016883
申请日：2002-05-24
申请人： ISIS PHARMACEUTICALS, INC. , MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G.
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G.
IPC分类号： C12Q1/68
CPC分类号： C07K14/003 , C07K1/1077
摘要： The present peptide nucleic acids exhibit enhanced cellular uptake and distribution. The peptide nucleic acids of the invention comprise naturally-occurring nucleobases and non-naturally-occurring nucleobases attached to a polyamide backbone. Non-naturally-occurring bases include monocyclic, bi-cyclic, and tricyclic heterocycles. Modified backbones are also provided.
摘要翻译：本发明的肽核酸表现出增强的细胞摄取和分布。本发明的肽核酸包含天然存在的核碱基和连接到聚酰胺主链上的非天然存在的核碱基。非天然存在的碱包括单环，双环和三环杂环。还提供了修改后的主干。

16. 发明申请

WO2013163430A2 SERPINC1 IRNA COMPOSITIONS AND METHODS OF USE THEREOF 审中-公开
标题翻译： SERPINC1 IRNA组合物及其使用方法
公开(公告)号：WO2013163430A2
公开(公告)日：2013-10-31
申请号：PCT/US2013/038218
申请日：2013-04-25
申请人： ALNYLAM PHARMACEUTICALS , AKINC, Akin , SEHGAL, Alfica , TOUDJARSKA, Ivanka , FOSTER, Donald , MILSTEIN, Stuart , BETTENCOURT, Brian , MAIER, Martin , CHARISSE, Klaus , KUCHIMANCHI, Satyanarayana , RAJEEV, Kallanthottathil, G. , MANOHARAN, Muthiah
发明人： AKINC, Akin , SEHGAL, Alfica , TOUDJARSKA, Ivanka , FOSTER, Donald , MILSTEIN, Stuart , BETTENCOURT, Brian , MAIER, Martin , CHARISSE, Klaus , KUCHIMANCHI, Satyanarayana , RAJEEV, Kallanthottathil, G. , MANOHARAN, Muthiah
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K31/713 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/335 , C12N2310/351 , C12N2310/3515 , C12N2310/3521 , C12N2310/3533 , C12N2320/30 , C12N2310/3525 , C12N2310/3523
摘要： The invention relates to iRNA, e.g., double- stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and methods of treating subjects having a bleeding disorder, such as a hemophilia.
摘要翻译：本发明涉及iRNA，例如双链核糖核酸（dsRNA），靶向Serpinc1基因的组合物和使用这种iRNA的方法，例如dsRNA，抑制Serpinc1表达的组合物和治疗出血性疾病的受试者的方法，如血友病。

17. 发明申请

WO2012037254A1 MODIFIED iRNA AGENTS 审中-公开
标题翻译：改性iRNA代理
公开(公告)号：WO2012037254A1
公开(公告)日：2012-03-22
申请号：PCT/US2011/051597
申请日：2011-09-14
申请人： ALNYLAM PHARMACEUTICALS, INC. , RAJEEV, Kallanthottathil, G. , ZIMMERMANN, Tracy , MANOHARAN, Muthiah , MAIER, Martin , FITZGERALD, Kevin
发明人： RAJEEV, Kallanthottathil, G. , ZIMMERMANN, Tracy , MANOHARAN, Muthiah , MAIER, Martin , FITZGERALD, Kevin
IPC分类号： C12N15/113 , A61K31/713 , C12N15/87 , C07H21/00
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。

18. 发明申请

WO2011153493A3 BIODEGRADABLE LIPIDS FOR THE DELIVERY OF ACTIVE AGENTS 审中-公开
标题翻译：用于交付活性剂的生物可降解脂肪
公开(公告)号：WO2011153493A3
公开(公告)日：2012-01-19
申请号：PCT/US2011039164
申请日：2011-06-03
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , MAIER MARTIN , JAYARAMAN MUTHUSAMY , MATSUDA SHIGEO , JAYAPRAKASH NARAYANANNAIR K , RAJEEV KALLANTHOTTATHIL G
发明人： MANOHARAN MUTHIAH , MAIER MARTIN , JAYARAMAN MUTHUSAMY , MATSUDA SHIGEO , JAYAPRAKASH NARAYANANNAIR K , RAJEEV KALLANTHOTTATHIL G
IPC分类号： A61K9/10 , A61K39/00 , A61K47/48 , A61K48/00 , C07C69/02 , C07C211/62 , C07C211/63 , C12N15/87
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。

19. 发明申请

WO2011153493A2 BIODEGRADABLE LIPIDS FOR THE DELIVERY OF ACTIVE AGENTS 审中-公开
标题翻译：用于交付活性剂的生物可降解脂肪
公开(公告)号：WO2011153493A2
公开(公告)日：2011-12-08
申请号：PCT/US2011/039164
申请日：2011-06-03
申请人： ALNYLAM PHARMACEUTICALS, INC. , MANOHARAN, Muthiah , MAIER, Martin , JAYARAMAN, Muthusamy , MATSUDA, Shigeo , JAYAPRAKASH, Narayanannair, K. , RAJEEV, Kallanthottathil, G.
发明人： MANOHARAN, Muthiah , MAIER, Martin , JAYARAMAN, Muthusamy , MATSUDA, Shigeo , JAYAPRAKASH, Narayanannair, K. , RAJEEV, Kallanthottathil, G.
IPC分类号： A61K47/44 , C07C229/30 , A61K31/713 , A61K31/7088 , C12N5/071 , C12N15/85 , A61P37/04
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。

20. 发明申请

WO2011143230A1 METHODS AND COMPOSITIONS FOR DELIVERY OF ACTIVE AGENTS 审中-公开
标题翻译：用于递送活性剂的方法和组合物
公开(公告)号：WO2011143230A1
公开(公告)日：2011-11-17
申请号：PCT/US2011/035939
申请日：2011-05-10
申请人： ALNYLAM PHARMACEUTICALS , MANOHARAN, Muthiah , RAJEEV, Kallanthottathil G.
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottathil G.
IPC分类号： A61K9/127 , A61K48/00
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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