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1. 发明申请

WO2006122053A3 BIOMARKERS FOR EVALUATING LIKELIHOOD OF TUMOR SENSITIVITY TO AN MTOR INHIBITOR 审中-公开
标题翻译：用于评估肿瘤抑制剂的肿瘤敏感性的生物标志物
公开(公告)号：WO2006122053A3
公开(公告)日：2009-04-30
申请号：PCT/US2006017770
申请日：2006-05-08
申请人： ARIAD GENE THERAPEUTICS INC , BEDROSIAN CAMILLE L , CLACKSON TIMOTHY P , RIVERA VICTOR
发明人： BEDROSIAN CAMILLE L , CLACKSON TIMOTHY P , RIVERA VICTOR
IPC分类号： C12Q1/00
CPC分类号： C12Q1/6886 , C12Q2600/106 , C12Q2600/136 , G01N33/57484 , G01N2800/52
摘要： The present invention provides compositions and methods for evaluating the likelihood that a tumor is sensitive to an mTOR inhibitor, e.g., rapamycin or a rapamycin analog. The invention provides FKBP proteins as biomarkers for predicting the likelihood that a tumor is sensitive to an mTOR inhibitor. The methods include assessing the expression or activity of an FKBP protein, e.g., FKBP 12, in a subject with a tumor or in a sample derived from a tumor. Additional biomarkers and biomarker combinations are also provided. The invention also provides kits containing, e.g., a validated antibody or ligand for assessing the expression or activity of an FKBP protein.
摘要翻译：本发明提供用于评价肿瘤对mTOR抑制剂例如雷帕霉素或雷帕霉素类似物敏感的可能性的组合物和方法。本发明提供FKBP蛋白作为生物标志物，用于预测肿瘤对mTOR抑制剂敏感的可能性。所述方法包括在具有肿瘤的受试者或衍生自肿瘤的样品中评估FKBP蛋白（例如FKBP 12）的表达或活性。还提供了额外的生物标志物和生物标志物组合。本发明还提供了包含例如用于评估FKBP蛋白的表达或活性的经验证的抗体或配体的试剂盒。

2. 发明申请

WO2007126798A3 REGULATED EXPRESSION OF RECOMBINANT PROTEINS FROM ADENO-ASSOCIATED VIRAL VECTORS 审中-公开
标题翻译：来自ADENO相关病毒载体的重组蛋白的调控表达
公开(公告)号：WO2007126798A3
公开(公告)日：2008-10-09
申请号：PCT/US2007007542
申请日：2007-03-29
申请人： CELL GENESYS INC , ARIAD PHARMA INC , HARDING THOMAS C , FANG JIANMIN , RIVERA VICTOR M , MINH NGUYEN , CLACKSON TIMOTHY P , JOOSS KARIN
发明人： HARDING THOMAS C , FANG JIANMIN , RIVERA VICTOR M , MINH NGUYEN , CLACKSON TIMOTHY P , JOOSS KARIN
IPC分类号： A61K48/00 , C07H21/02 , C07H21/04
CPC分类号： C12N15/86 , A61K48/0066 , C07K14/005 , C07K16/2863 , C07K2319/50 , C12N2750/14143 , C12N2770/32122 , C12N2830/002
摘要： Single AAV vector constructs for regulated expression of an immunoglobulin molecule or fragment thereof and methods of making and using the same are described. The AAV vectors comprise a regulated promoter operably linked to the coding sequence for a first and second immunoglobulin coding sequence, a sequence encoding a self-processing cleavage site between the coding sequence for the first and second immunoglobulin coding sequence and a additional proteolytic cleavage site, which provides a means to remove the self processing peptide sequenc from an expressed immunoglobulin molecule or fragment thereof. The vector constructs find utility in enhanced production of biologically active immunoglobulins or fragments thereof in vitro and in vivo.
摘要翻译：描述了免疫球蛋白分子或其片段的调节表达的单一AAV载体构建体及其制备和使用方法。 AAV载体包含与第一和第二免疫球蛋白编码序列的编码序列可操作地连接的调控启动子，编码第一和第二免疫球蛋白编码序列的编码序列与另外的蛋白水解切割位点之间的自加工切割位点的序列，其提供了从表达的免疫球蛋白分子或其片段中除去自身加工肽序列的方法。载体构建体在体外和体内发现在生物活性免疫球蛋白或其片段的增强生产中起作用。

3. 发明申请

WO2006122053A2 BIOMARKERS FOR EVALUATING LIKELIHOOD OF TUMOR SENSITIVITY TO AN MTOR INHIBITOR 审中-公开
标题翻译：用于评估肿瘤抑制剂的肿瘤敏感性的生物标志物
公开(公告)号：WO2006122053A2
公开(公告)日：2006-11-16
申请号：PCT/US2006/017770
申请日：2006-05-08
申请人： ARIAD GENE THERAPEUTICS, INC. , BEDROSIAN, Camille, L. , CLACKSON, Timothy, P. , RIVERA, Victor
发明人： BEDROSIAN, Camille, L. , CLACKSON, Timothy, P. , RIVERA, Victor
IPC分类号： C12N15/09
CPC分类号： C12Q1/6886 , C12Q2600/106 , C12Q2600/136 , G01N33/57484 , G01N2800/52
摘要： The present invention provides compositions and methods for evaluating the likelihood that a tumor is sensitive to an mTOR inhibitor, e.g., rapamycin or a rapamycin analog. The invention provides FKBP proteins as biomarkers for predicting the likelihood that a tumor is sensitive to an mTOR inhibitor. The methods include assessing the expression or activity of an FKBP protein, e.g., FKBP 12, in a subject with a tumor or in a sample derived from a tumor. Additional biomarkers and biomarker combinations are also provided. The invention also provides kits containing, e.g., a validated antibody or ligand for assessing the expression or activity of an FKBP protein.
摘要翻译：本发明提供用于评价肿瘤对mTOR抑制剂例如雷帕霉素或雷帕霉素类似物敏感的可能性的组合物和方法。本发明提供FKBP蛋白作为生物标志物，用于预测肿瘤对mTOR抑制剂敏感的可能性。所述方法包括在具有肿瘤的受试者或衍生自肿瘤的样品中评估FKBP蛋白（例如FKBP 12）的表达或活性。还提供了额外的生物标志物和生物标志物组合。本发明还提供了包含例如用于评估FKBP蛋白的表达或活性的经验证的抗体或配体的试剂盒。

4. 发明申请

WO0023602A3 REGULATED EXPRESSION USING CONDITIONAL ER RETENTION DOMAINS 审中-公开
标题翻译：使用条件保留域的调节表达式
公开(公告)号：WO0023602A3
公开(公告)日：2001-08-02
申请号：PCT/US9924327
申请日：1999-10-19
申请人： ARIAD GENE THERAPEUTICS INC , RIVERA VICTOR , CLACKSON TIMOTHY , ROTHMAN JAMES
发明人： RIVERA VICTOR , CLACKSON TIMOTHY , ROTHMAN JAMES
IPC分类号： A01K67/027 , C07K14/47 , C07K14/62 , C07K14/81 , C07K19/00 , C12N5/10 , C12N15/09 , C12N15/62 , C12P21/02 , C12P21/08 , C12Q1/02 , G01N33/15 , G01N33/50 , G01N33/68 , C12N15/63 , C12N15/67
CPC分类号： C07K14/62 , A01K2217/05 , C07K14/47 , C07K14/4702 , C07K14/811 , C07K2319/00 , C07K2319/03 , C07K2319/04 , C07K2319/09 , C07K2319/32 , C07K2319/50 , C07K2319/60 , C07K2319/71 , C07K2319/715 , C07K2319/75 , C07K2319/81 , C12N15/625 , C12N2799/022
摘要： Materials and methods involving conditional retention domains (CRDs) are disclosed. A conditional retention domain is any domain which is retained in the ER or other secretory compartment in the absence of ligand and is released from the secretory machinery when ligand is bound, i.e. in the presence of ligand. Also disclosed are fusion proteins containing CRDs and cells expressing such fusion proteins. In addition, the invention provides novel methods for producing target proteins in vivo using fusion proteins containing conditional retention domains and methods for identifying novel CRDs.
摘要翻译：公开了涉及条件保留域（CRDs）的材料和方法。条件保留结构域是在不存在配体的情况下保留在ER或其他分泌室中的任何结构域，并且当配体结合时，即在配体存在下，从分泌机制释放。还公开了含有CRD和表达这种融合蛋白的细胞的融合蛋白。此外，本发明提供了使用含有条件保留结构域的融合蛋白体内产生靶蛋白质的新方法和鉴定新型CRD的方法。

5. 发明申请

WO2011053938A8 METHODS AND COMPOSITIONS FOR TREATING CANCER 审中-公开
标题翻译：治疗癌症的方法和组合物
公开(公告)号：WO2011053938A8
公开(公告)日：2011-08-11
申请号：PCT/US2010055016
申请日：2010-11-01
申请人： ARIAD PHARMA INC , HUANG WEI-SHENG , RIVERA VICTOR M , CLACKSON TIMOTHY P , SHAKESPEARE WILLIAM C , SQUILLACE RACHEL M , GOZGIT JOSEPH M
发明人： HUANG WEI-SHENG , RIVERA VICTOR M , CLACKSON TIMOTHY P , SHAKESPEARE WILLIAM C , SQUILLACE RACHEL M , GOZGIT JOSEPH M
IPC分类号： A61K31/00 , A61K31/535 , A61K31/55
CPC分类号： C07D487/04 , A61K31/5025
摘要： The invention features methods, kits, and pharmaceutical compositions for treating cancer using 3-(imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-(4-((4-methylpiperazin-1-y1)-methyl)-3-(trifluoromethyl)phenyl)benzamide.
摘要翻译：（咪唑并[1,2-b]哒嗪-3-基乙炔基）-4-甲基-N-（4 - （（4-甲基哌嗪-1-基）） - 甲基）-3-（三氟甲基）苯基）苯甲酰胺。

6. 发明申请

WO2006063247A3 REGULATED EXPRESSION OF TRANSGENES IN THE CENTRAL NERVOUS SYSTEM OF MAMMALS 审中-公开
公开(公告)号：WO2006063247A3
公开(公告)日：2006-06-15
申请号：PCT/US2005/044654
申请日：2005-12-09
申请人： AVIGEN, INC. , SANFTNER, Laura, McGee , RIVERA, Victor, M.
发明人： SANFTNER, Laura, McGee , RIVERA, Victor, M.
IPC分类号： C12N15/864 , A61K48/00 , A61P25/16
摘要： Recombinant adeno-associated virus (rAAV) vectors are provided for delivery of regulatable transgenes to the central nervous system (CNS) of a mammal. Also provided are methods of treatment of subjects with neurodegenerative disorders using the vectors, and kits for constructing or using the vectors or performing the methods of the invention. Transgene sequences are expressed from a promoter/enhancer region comprising one or more binding sites for a transcription factor that is responsive to a small molecule inducer. Both the transgene construct and a construct comprising the transcription factor are delivered to the target cells. The regulatable transgene may be delivered on the same rAAV vector as the transcription factor, or on a separate vector. The transcription factor may comprise two polypeptide chains, e.g. a DNA binding domain and a transcription activation domain, that form an active dimer in the presence of a dimerizer such as rapamycin or a non-immunogenic analog thereof. Vectors, methods and kits of the invention may be used to deliver genes such as AADC or GDNF to the brain of a subject with a neurodegenerative disorder, such as Parkinson's disease, where the expression of AADC or GDNF in the brain can subsequently be regulated by treatment of the subject with rapamycin or a rapamycin analog.

7. 发明申请

WO0142444A3 METHODS FOR EXPRESSION OF GENES IN PRIMATES 审中-公开
标题翻译：用于表达前体基因的方法
公开(公告)号：WO0142444A3
公开(公告)日：2002-01-10
申请号：PCT/US0033256
申请日：2000-12-08
申请人： ARIAD GENE THERAPEUTICS INC , UNIV PENNSYLVANIA , RIVERA VICTOR , ZOLTICK PHILIP , WILSON JAMES M
发明人： RIVERA VICTOR , ZOLTICK PHILIP , WILSON JAMES M
IPC分类号： C12N15/09 , A01K67/027 , A61K31/7088 , A61K35/76 , A61K39/00 , A61K48/00 , C07K14/505 , C12N5/10 , C12N15/85 , C12N15/864 , C12N15/90
CPC分类号： A01K67/0275 , A01K2217/05 , A61K39/00 , A61K48/00 , C07K14/505 , C07K2319/00 , C12N15/85 , C12N15/86 , C12N2750/14143 , C12N2830/003 , C12N2830/006 , C12N2840/203
摘要： Methods for high level expression of genes in primates are disclosed. Such methods involve expression of transgenes comprising an RSV promoter and a nucleic acid sequence that is heterologous thereto.
摘要翻译：公开了在灵长类动物中高水平表达基因的方法。这样的方法涉及包含RSV启动子和与其异源的核酸序列的转基因的表达。

8. 发明申请

WO2007120298A3 METHODS, SUBSTRATES AND PROBES FOR ASSAYING N-GLYCOSIDASE ACTIVITY 审中-公开
标题翻译：方法，用于测定N-糖苷酶活性的基质和探针
公开(公告)号：WO2007120298A3
公开(公告)日：2008-06-05
申请号：PCT/US2006061332
申请日：2006-11-29
申请人： US ARMY MED RES MAT COMMAND , KEENER WILLIAM K , POLI MARK A , RIVERA VICTOR R
发明人： KEENER WILLIAM K , POLI MARK A , RIVERA VICTOR R
IPC分类号： G01N33/566 , C12Q1/68
CPC分类号： C12Q1/34
摘要： Disclosed herein are methods for assaying the RNA N-glycosidase or DNA N-glycosylase activity of protein. Also disclosed are substrates and probes that may be used in the assays.
摘要翻译：本文公开了用于测定蛋白质的RNA N-糖苷酶或DNA N-糖基化酶活性的方法。还公开了可用于测定中的底物和探针。

9. 发明申请

WO2006063247A2 REGULATED EXPRESSION OF TRANSGENES IN THE CENTRAL NERVOUS SYSTEM OF MAMMALS 审中-公开
标题翻译：在中央神经系统中的转移的调控表达
公开(公告)号：WO2006063247A2
公开(公告)日：2006-06-15
申请号：PCT/US2005044654
申请日：2005-12-09
申请人： AVIGEN INC , SANFTNER LAURA MCGEE , RIVERA VICTOR M
发明人： SANFTNER LAURA MCGEE , RIVERA VICTOR M
IPC分类号： C12N15/864 , A61K48/00 , A61P25/16
CPC分类号： C12N15/86 , A61K48/00 , C12N2750/14143 , C12N2830/00 , C12N2830/42 , C12N2830/85 , C12N2840/203
摘要： Recombinant adeno-associated virus (rAAV) vectors are provided for delivery of regulatable transgenes to the central nervous system (CNS) of a mammal. Also provided are methods of treatment of subjects with neurodegenerative disorders using the vectors, and kits for constructing or using the vectors or performing the methods of the invention. Transgene sequences are expressed from a promoter/enhancer region comprising one or more binding sites for a transcription factor that is responsive to a small molecule inducer. Both the transgene construct and a construct comprising the transcription factor are delivered to the target cells. The regulatable transgene may be delivered on the same rAAV vector as the transcription factor, or on a separate vector. The transcription factor may comprise two polypeptide chains, e.g. a DNA binding domain and a transcription activation domain, that form an active dimer in the presence of a dimerizer such as rapamycin or a non-immunogenic analog thereof. Vectors, methods and kits of the invention may be used to deliver genes such as AADC or GDNF to the brain of a subject with a neurodegenerative disorder, such as Parkinson's disease, where the expression of AADC or GDNF in the brain can subsequently be regulated by treatment of the subject with rapamycin or a rapamycin analog.
摘要翻译：提供重组腺相关病毒（rAAV）载体用于将可调节转基因递送至哺乳动物的中枢神经系统（CNS）。还提供了使用载体治疗患有神经变性疾病的受试者的方法，以及用于构建或使用载体或执行本发明的方法的试剂盒。转基因序列由启动子/增强子区表达，所述启动子/增强子区包含对小分子诱导物有反应的转录因子的一个或多个结合位点。转基因构建体和包含转录因子的构建体均被递送至靶细胞。可调节的转基因可以在与转录因子相同的rAAV载体上递送，或者在单独的载体上递送。转录因子可以包含两条多肽链，例如 DNA结合结构域和转录激活结构域，其在二聚体如雷帕霉素或其非免疫原性类似物的存在下形成活性二聚体。可以使用本发明的载体，方法和试剂盒将诸如AADC或GDNF的基因递送至具有神经退行性疾病如帕金森病的受试者的脑中，其中脑中AADC或GDNF的表达随后可由用雷帕霉素或雷帕霉素类似物治疗受试者。

10. 发明申请

WO2013162727A1 METHODS AND COMPOSITIONS FOR RAF KINASE MEDIATED DISEASES 审中-公开
标题翻译：用于RAF激酶介导性疾病的方法和组合
公开(公告)号：WO2013162727A1
公开(公告)日：2013-10-31
申请号：PCT/US2013/030444
申请日：2013-03-12
申请人： ARIAD PHARMACEUTICALS, INC. , GOZGIT, Joseph, M. , RIVERA, Victor, M. , SHAKESPEARE, William, C. , ZHU, Xiaotian , DALGARNO, David, C.
发明人： GOZGIT, Joseph, M. , RIVERA, Victor, M. , SHAKESPEARE, William, C. , ZHU, Xiaotian , DALGARNO, David, C.
IPC分类号： A61K31/165
CPC分类号： C07D487/04 , A61K31/437 , A61K31/496 , A61K31/4985 , A61K31/5025 , A61K31/5377 , C07D471/04 , C07D473/34
摘要： The invention discloses methods and compositions for treating or preventing RAF kinase mediated diseases or conditions by administering a compound of Formula 1: or a pharmaceutically acceptable salt, solvate or hydrate thereof, wherein the variables are defined as herein.
摘要翻译：本发明公开了通过施用式1化合物或其药学上可接受的盐，溶剂合物或水合物来治疗或预防RAF激酶介导的疾病或病症的方法和组合物，其中变量如本文所定义。

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