公开(公告)号：EP3080143A4

公开(公告)日：2017-06-14

申请号：EP14869305

申请日：2014-12-09

申请人： SANGAMO BIOSCIENCES INC

发明人： MILLER JEFFREY C ,  PASCHON DAVID ,  REBAR EDWARD J

IPC分类号： C07K14/00 ,  C12N9/22 ,  C12N9/64 ,  C12N15/09 ,  C12N15/62 ,  C12N15/86

CPC分类号： C07K14/47 ,  A61K48/00 ,  C07K14/4702 ,  C07K2319/81 ,  C12N9/22 ,  C12N9/644 ,  C12N15/86 ,  C12N2750/14133 ,  C12N2750/14142 ,  C12N2750/14143

摘要： Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins involved in clotting into the genome of a cell for treating conditions including hemophilias.

摘要翻译： 本文公开了用于插入转基因序列的方法和组合物，所述转基因序列编码涉及凝结成细胞基因组的蛋白质以治疗包括血友病的病症。

公开(公告)号：EP2424991A4

公开(公告)日：2012-11-14

申请号：EP10772363

申请日：2010-04-27

申请人： GENZYME CORP

发明人： PASSINI MARCO A ,  SHIHABUDDIN LAMYA ,  CHENG SENG H

IPC分类号： C12N15/79 ,  A61K31/7088 ,  A61K35/76 ,  C07K14/475 ,  C12N15/864

CPC分类号： A61K38/1709 ,  A61K31/7088 ,  A61K48/00 ,  A61K48/0008 ,  A61K48/005 ,  A61K48/0075 ,  C07K14/4702 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14171

摘要： Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

公开(公告)号：EP2176283A4

公开(公告)日：2011-06-08

申请号：EP08781733

申请日：2008-07-11

申请人： UNIV IOWA RES FOUND ,  DAVIDSON BEVERLY L ,  CHEN YONG HONG

发明人： DAVIDSON BEVERLY L ,  CHEN YONG HONG

IPC分类号： C07K14/025 ,  A61K48/00 ,  C12N15/00 ,  C12N15/34

CPC分类号： A61K38/162 ,  A61K48/00 ,  C07K5/08 ,  C07K5/10 ,  C07K7/06 ,  C07K14/005 ,  C07K2319/33 ,  C12N15/86 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/40 ,  G01N33/5058

摘要： The present disclosure provides targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents to the brain.

公开(公告)号：EP3132043A2

公开(公告)日：2017-02-22

申请号：EP15715750.4

申请日：2015-04-15

申请人： Universitätsklinikum Hamburg-Eppendorf

发明人： KÖRBELIN, Jakob ,  MICHELFELDER, Stefan ,  TREPEL, Martin

IPC分类号： C12N15/861

CPC分类号： C07K7/06 ,  A61K38/00 ,  A61K48/00 ,  C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  C12N2710/14144 ,  C12N2750/14122 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2810/6027

摘要： The invention relates to novel peptides, polypeptides or proteins which bind specifically to brain cells and/or to the spinal cord. The peptides, polypeptides, or proteins can be components of a viral capsid and can be used to lead a recombinant viral vector selectively to the brain and/or spinal cord after systemic administration to a subject and to ensure tissue-specific expression of one or more transgenes there. The invention also relates to a recombinant viral vector, preferably an AAV vector, which comprises a capsid containing at least one of the claimed peptides, polypeptides, or proteins and which comprises at least one transgene packaged in the capsid. Said viral vector can be used, in particular for the therapeutic treatment of a disease or disorder of the brain and/or spinal cord. The invention further relates to cells and pharmaceutical compositions that comprise the viral vector according to the invention.

摘要翻译： 本发明涉及特异性结合脑细胞和/或脊髓的新型肽，多肽或蛋白质。 肽，多肽或蛋白质可以是病毒衣壳的组分，并且可以用于在对受试者进行系统给药之后选择性地将重组病毒载体引导至脑和/或脊髓，并确保一种或多种的组织特异性表达 转基因在那里 本发明还涉及重组病毒载体，优选AAV载体，其包含含有所要求保护的肽，多肽或蛋白质中的至少一种的衣壳，并且包含至少一种包装在衣壳中的转基因。 所述病毒载体可用于特别是治疗性治疗脑和/或脊髓疾病或病症。 本发明还涉及包含根据本发明的病毒载体的细胞和药物组合物。

公开(公告)号：EP3121190A1

公开(公告)日：2017-01-25

申请号：EP16186573.8

申请日：2008-07-11

申请人： University of Iowa Research Foundation ,  Davidson, Beverly L. ,  Chen, Yong Hong

发明人： DAVIDSON, Beverly L. ,  CHEN, Yong Hong

IPC分类号： C07K14/025 ,  A61K48/00 ,  C12N15/00 ,  C12N15/34

CPC分类号： A61K38/162 ,  A61K48/00 ,  C07K5/08 ,  C07K5/10 ,  C07K7/06 ,  C07K14/005 ,  C07K2319/33 ,  C12N15/86 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/40 ,  G01N33/5058

摘要： The present disclosure provides targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents to the brain.

摘要翻译： 本公开提供了靶向肽和载体，其含有编码将药物递送至脑的靶向肽的序列。

公开(公告)号：EP3030664A1

公开(公告)日：2016-06-15

申请号：EP14753039.8

申请日：2014-08-06

申请人： Universitätsklinikum Hamburg-Eppendorf

发明人： TREPEL, Martin ,  KOERBELIN, Jakob ,  MICHELFELDER, Stefan

IPC分类号： C12N15/86 ,  C12N15/10 ,  A61K48/00

CPC分类号： A61K38/162 ,  C07K14/005 ,  C07K2319/33 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2799/025 ,  C12N2810/40

摘要： The invention relates to a peptide, polypeptide, or protein that binds specifically to cells of the lung endothelium. The peptide, polypeptide, or protein can be a component of a viral capsid and can be used to lead a recombinant viral vector selectively to the lung endothelial tissue after systemic administration to a subject and to ensure tissue-specific expression of one or more transgenes there. The invention thus further relates to a recombinant viral vector, preferably an AAV vector, which comprises a capsid containing the peptide, polypeptide, or protein according to the invention and which comprises at least one transgene packaged in the capsid. The viral vector is suitable in particular for the therapeutic treatment of a lung disorder or a lung disease. The invention further relates to cells and pharmaceutical compositions that comprise the viral vector according to the invention.

公开(公告)号：EP2826860A1

公开(公告)日：2015-01-21

申请号：EP14181861.7

申请日：2011-04-22

申请人： The University of Massachusetts

发明人： Gao, Guangping ,  Zhang, Hongwei ,  Wang, Hongyan ,  Xu, Zuoshang

IPC分类号： C12N15/00 ,  C12N15/86 ,  A61K48/00

CPC分类号： C12N15/86 ,  A61K31/713 ,  A61K38/50 ,  A61K48/00 ,  A61K48/0058 ,  A61K48/0075 ,  C12N7/00 ,  C12N9/80 ,  C12N15/1137 ,  C12N15/635 ,  C12N15/8645 ,  C12N2310/141 ,  C12N2750/14133 ,  C12N2750/14141 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14162 ,  C12N2810/10 ,  C12N2840/007 ,  C12Y305/01015

摘要： The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.

摘要翻译： 本发明在一些方面涉及可用于靶向转基因到CNS组织的重组腺相关病毒，以及包含其的组合物及其使用方法。 在一些方面，本发明提供了用于治疗CNS相关疾病的方法和组合物。

公开(公告)号：EP2826860B1

公开(公告)日：2018-08-22

申请号：EP14181861.7

申请日：2011-04-22

申请人： University of Massachusetts

发明人： Gao, Guangping ,  Zhang, Hongwei ,  Wang, Hongyan ,  Xu, Zuoshang

IPC分类号： C12N15/00 ,  C12N15/86 ,  A61K48/00

CPC分类号： C12N15/86 ,  A61K31/713 ,  A61K38/50 ,  A61K48/00 ,  A61K48/0058 ,  A61K48/0075 ,  C12N7/00 ,  C12N9/80 ,  C12N15/1137 ,  C12N15/635 ,  C12N15/8645 ,  C12N2310/141 ,  C12N2750/14133 ,  C12N2750/14141 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14162 ,  C12N2810/10 ,  C12N2840/007 ,  C12Y305/01015

摘要： The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.

公开(公告)号：EP2176283B1

公开(公告)日：2016-11-02

申请号：EP08781733.4

申请日：2008-07-11

申请人： University of Iowa Research Foundation ,  Davidson, Beverly L. ,  Chen, Yong Hong

发明人： DAVIDSON, Beverly, L. ,  CHEN, Yong Hong

IPC分类号： C07K14/025 ,  A61K48/00 ,  C12N15/00 ,  C12N15/34

CPC分类号： A61K38/162 ,  A61K48/00 ,  C07K5/08 ,  C07K5/10 ,  C07K7/06 ,  C07K14/005 ,  C07K2319/33 ,  C12N15/86 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/40 ,  G01N33/5058

摘要： The present disclosure provides targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents to the brain.

公开(公告)号：EP2561073B1

公开(公告)日：2016-08-24

申请号：EP11772784.2

申请日：2011-04-22

申请人： University of Massachusetts

发明人： GAO, Guangping ,  ZHANG, Hongwei ,  WANG, Hongyan ,  XU, Zuoshang

IPC分类号： C12N15/00 ,  C12N15/86 ,  A61K48/00

CPC分类号： C12N15/86 ,  A61K31/713 ,  A61K38/50 ,  A61K48/00 ,  A61K48/0058 ,  A61K48/0075 ,  C12N7/00 ,  C12N9/80 ,  C12N15/1137 ,  C12N15/635 ,  C12N15/8645 ,  C12N2310/141 ,  C12N2750/14133 ,  C12N2750/14141 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14162 ,  C12N2810/10 ,  C12N2840/007 ,  C12Y305/01015

摘要： The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.