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公开(公告)号：EP2855679B1
公开(公告)日：2018-07-18
申请号：EP13733463.7
申请日：2013-05-30
申请人： QBI Enterprises Ltd. , Bio-Lab Ltd.
发明人： AVKIN-NACHUM, Sharon , TRUFFERT, Jean-Christophe , LEFOIX, Myriam , HILDESHEIM, Jean , KLEINMAN, Tirtsa
IPC分类号： C12N15/113 , A61K31/7115
CPC分类号： C12N15/113 , A61K31/7115 , C12N2310/14 , C12N2310/314 , C12N2310/321 , C12N2310/333 , C12N2310/336 , C12N2310/344 , C12N2320/51 , C12N2320/53 , C12N2330/30 , C12N2310/3521
摘要： Disclosed herein are double-stranded RNA nucleic acid molecules, which include at least one pyrazolotriazine nucleotide analog and have been modified to exhibit one of the following, increased on-target activity, increased target specificity, enhanced nuclease stability, reduced off target activity and/or reduced immunogenicity when compared to an unmodified or similarly modified dsRNA; pharmaceutical compositions comprising such molecules and methods of use thereof in therapy.
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公开(公告)号：EP3227447A4
公开(公告)日：2018-07-11
申请号：EP15866342
申请日：2015-12-03
IPC分类号： C12N15/113 , C07H21/00 , C12N9/22
CPC分类号： C12N15/111 , C07H21/02 , C12N9/22 , C12N15/113 , C12N15/907 , C12N2310/10 , C12N2310/20 , C12N2310/312 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/3231 , C12N2310/333 , C12N2310/335 , C12N2310/346 , C12N2310/3517 , C12N2310/531 , C12N2320/51 , C12N2320/53 , C12N2330/31 , C12Q1/6876 , C12N2310/3521 , C12N2310/3533
摘要： The present invention relates to modified guide RNAs and their use in clustered, regularly interspaced, short palindromic repeats (CRISPR)/CRISPR-associated (Cas) systems.
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3.发明公开RNA MODULATING OLIGONUCLEOTIDES WITH IMPROVED CHARACTERISTICS FOR THE TREATMENT OF NEUROMUSCULAR DISORDERS 审中-公开RNA调节具有改善特性的寡核苷酸治疗神经肌肉疾病
公开(公告)号：EP3228712A1
公开(公告)日：2017-10-11
申请号：EP17170330.9
申请日：2013-04-23
IPC分类号： C12N15/113 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61P25/00
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/333 , C12N2310/334 , C12N2310/3341 , C12N2310/335 , C12N2310/336 , C12N2310/346 , C12N2310/3521 , C12N2320/34
摘要： The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.
摘要翻译： 本发明提供了改进的寡核苷酸及其用于治疗，改善，预防，延缓和/或治疗人顺式元件重复不稳定性相关的遗传性神经肌肉疾病或神经变性疾病。
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公开(公告)号：EP2970971A4
公开(公告)日：2016-11-02
申请号：EP14770615
申请日：2014-03-13
申请人： SHEKDAR KAMBIZ
发明人： SHEKDAR KAMBIZ
IPC分类号： C12N15/113 , C12N15/10
CPC分类号： C12N15/1138 , C12N15/102 , C12N2310/152 , C12N2310/3181 , C12N2310/3231 , C12N2310/333 , C12N2310/336 , C12N2310/533 , C12N2330/50 , C12Q1/6876
摘要： The invention provides compositions and methods of making and using effector oligonucleotides, including effector oligonucleotides with greater than one mismatch as compared to its target sequence. These effector oligonucleotides are useful for improving the efficiency of genomic editing as well as providing therapeutic benefits to individuals in need thereof.
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5.发明公开OLIGONUCLEOTIDE INHIBITORS WITH CHIMERIC BACKBONE AND 2-AMINO-2'-DEOXYADENOSINE 审中-公开用钥匙链嵌合和2-氨基-2'-脱氧腺苷OLIGONUKLEOTIDHEMMER
公开(公告)号：EP2678431A4
公开(公告)日：2015-07-15
申请号：EP12749352
申请日：2012-02-23
申请人： PHARMAXIS LTD
发明人： SEGUIN ROSANNE , FERRARI NICOLAY
IPC分类号： C12N15/113 , A61K31/7125 , A61P11/00 , A61P29/00 , C07H21/00 , C12N15/19 , C12N15/55
CPC分类号： C12N15/1137 , A61K31/7125 , C07H21/00 , C12N15/111 , C12N15/1136 , C12N15/1138 , C12N2310/11 , C12N2310/315 , C12N2310/333 , C12N2310/343 , C12Y301/04017
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6.发明公开DOUBLE-STRANDED OLIGONUCLEOTIDE COMPOUNDS FOR TREATING HEARING AND BALANCE DISORDERS 审中-公开用于治疗听力和平衡失调的双链寡核苷酸化合物
公开(公告)号：EP2739637A1
公开(公告)日：2014-06-11
申请号：EP12819429.7
申请日：2012-08-03
IPC分类号： C07H21/00 , C12N15/113 , A61K31/7088
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2310/321 , C12N2310/332 , C12N2310/333 , C12N2310/3341 , C12N2310/335 , C12N2310/336 , C12N2310/337 , C12N2310/3521 , C12N2310/50
摘要： The present application relates to double stranded nucleic acid compounds, compositions comprising same and methods of use thereof for the treatment of hearing loss in a subject in need thereof. The compounds are preferably chemically synthesized and modified dsRNA molecules which inhibit expression of a gene expressed selected from the group consisting of HES1, HES5, HEY1, HEY2, ID1, ID2, ID3, CDKN1B, and NOTCHl.
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公开(公告)号：EP1572902B1
公开(公告)日：2014-06-11
申请号：EP03708928.1
申请日：2003-01-31
IPC分类号： C12N15/11 , C12N15/113 , C12N5/10 , C12P19/34
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1135 , C12N15/1137 , C12N2310/111 , C12N2310/14 , C12N2310/322 , C12N2310/333 , C12N2310/334 , C12N2310/335 , C12N2310/336 , C12N2310/53 , C12N2320/50 , C12Y102/01012
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8.发明授权
公开(公告)号：EP1702620B1
公开(公告)日：2014-01-08
申请号：EP04799849.7
申请日：2004-11-19
IPC分类号： A61K31/7115 , A61K48/00 , C12N5/10 , A01K67/027 , A61P37/04
CPC分类号： C12N15/117 , A61K31/7115 , A61K39/39 , A61K2039/53 , A61K2039/55561 , C12N2310/333
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9.发明授权PETITS ARN INTERFERENTS SPECIFIQUES DES SOUS-UNITES ALPHA, ALPHA' ET ß DE LA PROTEINE KINASE CK2 ET LEURS APPLICATIONS 有权下α，α单位和蛋白激酶CK2特异性siRNA的BETA，以及它们的应用
公开(公告)号：EP1651762B1
公开(公告)日：2013-11-20
申请号：EP04767568.1
申请日：2004-07-02
申请人： Commissariat à l'Énergie Atomique et aux Énergies Alternatives , INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
IPC分类号： A61K38/00 , C12N15/11 , C12N15/63 , A61K31/713
CPC分类号： C12N15/1137 , A01K2217/05 , A61K31/713 , A61K38/00 , C12N2310/111 , C12N2310/14 , C12N2310/322 , C12N2310/33 , C12N2310/333 , C12N2310/334 , C12N2310/335 , C12Y207/11001
摘要： The invention relates to small interfering RNA specific to sub-units α, α′ and &bgr; of the kinase protein CK2, and to the applications of the same, especially for treating cancer and viral illnesses.
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公开(公告)号：EP1572902A2
公开(公告)日：2005-09-14
申请号：EP03708928.1
申请日：2003-01-31
申请人： AMBION, INC.
IPC分类号： C12N1/00
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1135 , C12N15/1137 , C12N2310/111 , C12N2310/14 , C12N2310/322 , C12N2310/333 , C12N2310/334 , C12N2310/335 , C12N2310/336 , C12N2310/53 , C12N2320/50 , C12Y102/01012
摘要： The present invention provides improved methods of attenuating gene expression through the phenomenon of RNA interference. The invention provides methods of synthesis of double stranded RNAs (dsRNAs) of increased potency for use as small interfering RNA (siRNA). Surprisingly and unexpectedly, siRNAs made by the methods of the invention are significantly more potent than previously available siRNAs.
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