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公开(公告)号：EP2791335B1
公开(公告)日：2018-11-14
申请号：EP12815856.5
申请日：2012-12-17
IPC分类号： C12N15/11 , C12N15/113 , A61K31/713
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/15 , C12N2310/3181 , C12N2310/3231 , C12N2310/341 , C12N2310/3513 , C12N2310/3515 , C12N2310/53 , C12N2320/52
摘要： A method of reducing the level of a transcription product in a cell comprising contacting with the cell a composition comprising a double-stranded nucleic acid complex comprising a first nucleic acid strand annealed to a second nucleic acid strand, wherein: (i) the first nucleic acid strand hybridizes to the transcription product and comprises (a) a region consisting of at least 4 consecutive nucleotides that are recognized by RNase H when the strand is hybridized to the transcription product, (b) one or more nucleotide analogs located on 5′ terminal side of the region, (c) one or more nucleotide analogs located on 3′ terminal side of the region and (d) a total number of nucleotides and nucleotide analogs ranging from 8 to 35 nucleotides and (ii) the second nucleic acid strand comprises (a) nucleotides and optionally nucleotide analogs and (b) at least 4 consecutive RNA nucleotides.
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公开(公告)号：EP2951305B1
公开(公告)日：2018-08-15
申请号：EP14704307.9
申请日：2014-01-30
发明人： ALBÆK, Nanna , HANSEN, Henrik Frydenlund , KAMMLER, Susanne , RAVN, Jacob , ØRUM, Henrik , TURNER, Mark , KRAMPERT, Monika , HADWIGER, Philipp , OTTOSEN, Søren , LINDOW, Morten
IPC分类号： C12N15/113 , A61K31/713 , A61K47/55
CPC分类号： C12N15/113 , A61K31/712 , A61K31/713 , A61K47/55 , A61K47/64 , C12N15/1131 , C12N15/1137 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/3231 , C12N2310/341 , C12N2310/351 , C12N2310/3513 , C12N2320/30 , C12N2330/30
摘要： The invention provides LNA therapeutics oligonucleotide carbohydrate conjugates with considerably enhanced potency, extended therapeutic index and reduced toxicity.
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公开(公告)号：EP3004351B1
公开(公告)日：2018-08-08
申请号：EP14807845.4
申请日：2014-06-04
申请人： Tribiotica Llc
发明人： DUNN, Ian , LAWLER, Matthew
CPC分类号： A61K47/549 , A61K39/0011 , A61K2039/5158 , A61K2039/6025 , C12N15/1068 , C12N15/111 , C12N15/1131 , C12N2310/351 , C12N2310/3513 , C12N2310/3517 , C12N2320/30 , C12Q1/6811 , C12N2310/321 , C12N2310/3521 , C12Q2543/10 , C12Q2523/109
摘要： The present disclosure is directed methods and products for synthesizing and using targeted templated assembly reactants comprising at least one nucleic acid recognition moiety, at least one selectively-reactive moiety, and at least one effector partial moiety. The nucleic acid recognition moiety can bind a target nucleic acid sequence within a sample. The nucleic acid recognition moiety also can bind the selectively-reactive moiety. Additionally, the effector partial moiety can bind the selectively-reactive moiety to produce an active effector structure. Also disclosed are methods of delivering the targeted templated assembly reactants and active effector structures formed from the targeted templated assembly reactants.
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公开(公告)号：EP2046964B1
公开(公告)日：2018-07-04
申请号：EP07796847.7
申请日：2007-07-11
CPC分类号： C12N15/87 , A61K48/00 , C12N15/111 , C12N2310/11 , C12N2310/315 , C12N2310/3513 , C12N2320/32 , C12N2330/30
摘要： This disclosure relates to nucleic acid constructs modified to have a reduced net anionic charge. In some aspects the constructs comprise phosphodiester and/or phosphothioate protecting groups. The disclosure also provide methods of making and using such constructs.
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公开(公告)号：EP3314027A1
公开(公告)日：2018-05-02
申请号：EP16818715.1
申请日：2016-06-29
申请人： Caris Science, Inc.
CPC分类号： C12N15/115 , A61K31/711 , A61K47/6807 , C12N2310/16 , C12N2310/3513 , C12N2320/10 , G01N33/53 , G01N2333/4716 , G01N2333/70503 , Y02A50/411
摘要： Methods and compositions are provided for oligonucleotides that bind targets of interest. The targets include cells and microvesicles, such as those derived from various diseases. The oligonucleotides can be used for diagnostic and therapeutic purposes. The target of the oligonucleotides can be a therapeutic target such as Complement Component 1, Q Subcomponent (C1q) or a subunit thereof.
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公开(公告)号：EP3297680A2
公开(公告)日：2018-03-28
申请号：EP16731346.9
申请日：2016-05-19
IPC分类号： A61K47/48
CPC分类号： A61K47/6843 , A61K45/06 , A61K47/6807 , A61K47/6849 , A61P35/00 , C07K16/18 , C12N15/1137 , C12N2310/14 , C12N2310/3513
摘要： A method of targeting a pharmaceutical agent to a senescent cell is disclosed. The method comprises administering the pharmaceutical agent to the subject, wherein said pharmaceutical agent is attached to an affinity moiety, said affinity moiety being capable of binding specifically to a polypeptide selected from the group consisting of HSP90B1, DNAJB4, PI4K2A, DBN1, PRKCSH, SPTBN1, NPM1, ITGA3 and a polypeptide set forth in Table 1. The targeting may be for therapeutics or diagnostics.
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公开(公告)号：EP3173482A4
公开(公告)日：2018-02-28
申请号：EP15818679
申请日：2015-07-08
申请人： UNIV KYOTO
发明人： SAITO HIROHIDE , OSADA ERIKO
IPC分类号： C12N15/09 , C07K19/00 , C12N15/113
CPC分类号： C07K19/00 , C07K16/32 , C07K2318/20 , C07K2319/00 , C12N15/09 , C12N15/113 , C12N2310/14 , C12N2310/3513
摘要： An RNA and protein delivery system utilizing an RNA-protein complex having a higher-order structure, and an RNA-protein complex having a novel higher-order structure.
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8.发明授权Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means 有权Duchenne型肌营养不良患者中有效外显子（44）跳过的方法及相关手段
公开(公告)号：EP2813570B1
公开(公告)日：2017-11-15
申请号：EP14179979.1
申请日：2009-05-14
发明人： Platenburg, Gerard Johannes , de Kimpe, Josephus Johannes , van Deutekom, Judith Christina Theodora , van Ommen, Garrit-Jan Boudewijn , Aartsma-Rus, Annemieke
IPC分类号： C12N15/11 , A61K31/7088 , C12N15/86
CPC分类号： C12N15/113 , A61K48/00 , C12N2310/11 , C12N2310/111 , C12N2310/314 , C12N2310/315 , C12N2310/321 , C12N2310/3233 , C12N2310/346 , C12N2310/3513 , C12N2310/3517 , C12N2320/33 , C12N2310/3521
摘要： The invention provides a method for modulating skipping of Duchenne Muscular Dystrophy (DMD) gene in a cell. More specifically, the invention provides methods and means for modulating skipping of exon 44 of DMD. The invention further provides nucleic acid molecules that can be used for a method of the invention, expression methods for expression of a nucleic acid molecule in a cell, and use of a nucleic acid molecule for modulating skipping of DMD in a cell.
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公开(公告)号：EP2735568B1
公开(公告)日：2017-08-02
申请号：EP13186973.7
申请日：2007-05-10
申请人： AVI BioPharma, Inc.
发明人： Weller, Dwight D. , Hassinger, Jed N. , Cai, Bao
CPC分类号： C07H21/00 , C12N15/111 , C12N15/1131 , C12N15/1132 , C12N15/1135 , C12N15/1137 , C12N15/1138 , C12N2310/3145 , C12N2310/3233 , C12N2310/351 , C12N2310/3513 , C12N2320/51 , C12N2330/30
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10.发明公开
公开(公告)号：EP3152308A2
公开(公告)日：2017-04-12
申请号：EP15803887.7
申请日：2015-06-08
发明人： BRADSHAW, Curt, W. , SAKAMURI, Sukumar , ELTEPU, Laxman , LAM, Son , LIU, Dingguo , MEADE, Bryan , IACONO, Giuseppe, Dello , STOCK, Joseph , LIU, Bin
CPC分类号： C12N15/113 , C07H19/10 , C07H19/20 , C07H21/02 , C07H21/04 , C12N15/85 , C12N2310/14 , C12N2310/31 , C12N2310/32 , C12N2310/346 , C12N2310/3513 , C12N2310/3515 , C12N2330/30
摘要： The invention features a hybridized polynucleotide construct containing a passenger strand, a guide strand loadable into a RISC complex, and (i) a 3′-terminal or an internucleotide non-bioreversible group in the guide strand; or (ii) a 5′-terminal, a 3′-terminal, or an internucleotide non-bioreversible group in the passenger strand, and a 5′-terminal, a 3′-terminal, or an internucleotide disulfide bioreversible group in the guide strand or the passenger strand. The invention also features methods of delivering a polynucleotide to a cell using the hybridized polynucleotide construct. The invention further features methods of reducing the expression of a polypeptide in a cell using the hybridized polynucleotide construct.
摘要翻译： 本发明的特征在于含有载体链，可加载到RISC复合物中的引导链和（i）引导链中的3'端或核苷酸非生物可逆组的杂交多核苷酸构建体; 或（ii）乘客链中的5'端，3'端或核苷酸非生物逆转基团，以及引物中的5'末端，3'末端或核苷酸间二硫键生物逆转组 绞线或乘客绳。 本发明还涉及使用杂交的多核苷酸构建体将多核苷酸递送至细胞的方法。 本发明还涉及使用杂交的多核苷酸构建体减少细胞中多肽表达的方法。
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