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1. 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。

2. 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。

3. 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.

4. 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。

5. 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。

6. 发明申请

WO2012051587A1 METHODS FOR INHIBITING CELL PROLIFERATION IN EGFR-DRIVEN CANCERS 审中-公开
标题翻译：抑制EGFR激活癌细胞增殖的方法
公开(公告)号：WO2012051587A1
公开(公告)日：2012-04-19
申请号：PCT/US2011/056457
申请日：2011-10-14
申请人： ARIAD PHARMACEUTICALS, INC. , DALGARNO, David, C. , SHAKEPEARE, William, C. , ZHU, Xiaotian , RIVERA, Victor, M. , MIRET, Juan, J.
发明人： DALGARNO, David, C. , SHAKEPEARE, William, C. , ZHU, Xiaotian , RIVERA, Victor, M. , MIRET, Juan, J.
IPC分类号： A01N43/54 , A61K31/505
CPC分类号： C07F9/65583 , A61K31/505 , C07D401/12 , C07F9/6561 , C07F9/6568
摘要： The invention features a method for treating patients who have an EGFR-driven cancer, which is, or has become, refractory to a tyrosine kinase inhibitor, such as eriotinib and gefitinib, by administering a compound of formula (I) to the patient. The invention also features treating EGFR-driven cancers having an EGFR mutation identified herein.
摘要翻译：本发明的特征在于通过向患者施用式（I）的化合物来治疗患有EGFR驱动的癌症的患者的方法，所述癌症已经或已经成为对酪氨酸激酶抑制剂诸如埃替替尼和吉非替尼难治的患者。本发明还特征在于治疗具有本文鉴定的EGFR突变的EGFR驱动的癌症。

7. 发明申请

WO2007126798A2 REGULATED EXPRESSION OF RECOMBINANT PROTEINS FROM ADENO-ASSOCIATED VIRAL VECTORS 审中-公开
标题翻译：从ADENO相关病毒载体调节重组蛋白的表达
公开(公告)号：WO2007126798A2
公开(公告)日：2007-11-08
申请号：PCT/US2007/007542
申请日：2007-03-29
申请人： CELL GENESYS, INC. , ARIAD PHARMACEUTICALS, 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： C07K16/00 , C12N15/00
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载体包含可操作地连接到第一和第二免疫球蛋白编码序列的编码序列的调节启动子，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可增强生物活性免疫球蛋白或其片段的生产。

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