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1.发明申请CAPSID-MODIFIED rAAV VECTOR COMPOSITIONS HAVING IMPROVED TRANSDUCTION EFFICIENCIES, AND METHODS OF USE 有权具有改进的传导效率的CAPSID改性的rAAV载体组合物及其使用方法公开(公告)号：US20140050701A1
公开(公告)日：2014-02-20
申请号：US13899481
申请日：2013-05-21
Inventor： Li Zhong , Sergei Zolotukhin , Lakshmanan Govindasamy , Mavis Agbandje-McKenna , Arun Srivastava
IPC: C07K14/005 , A61K48/00 , C12N15/86
CPC classification number: C07K14/005 , A61K35/76 , A61K39/0011 , A61K48/0008 , A61K48/005 , A61K48/0091 , A61K2039/5158 , C12N7/00 , C12N15/86 , C12N15/8645 , C12N2750/14122 , C12N2750/14132 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14171 , C12N2810/6027
Abstract: Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using novel capsid-protein-mutated rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more symptoms of disease or abnormal conditions via in situ and/or ex vivo mammalian gene therapy methods. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.
Abstract translation: 公开了衣壳修饰的rAAV表达载体，以及感染性病毒粒子，组合物和包括它们的药物制剂。 还公开了在各种诊断和治疗应用中制备和使用新型衣壳蛋白突变的rAAV载体构建体的方法，包括特别地，作为用于诊断，治疗或改善一种或多种疾病或异常状况症状的递送剂 通过原位和/或离体哺乳动物基因治疗方法。 还公开了用于衣壳修饰的rAAV表达载体，病毒颗粒和具有相对于未修饰的rAAV载体的转导效率具有改进的转导效率的感染性病毒粒子的大规模生产方法，以及在制造中使用所公开的组合物 的用于各种体外和/或体内应用的药物。
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2.发明申请
公开(公告)号：US20130203841A1
公开(公告)日：2013-08-08
申请号：US13855640
申请日：2013-04-02
Inventor： Li Zhong , Sergei Zolotukhin , Lakshmanan Govindasamy , Mavis Agbandje-McKenna , Arun Srivastava
IPC: A61K48/00
CPC classification number: C07K14/005 , A61K35/76 , A61K39/0011 , A61K48/0008 , A61K48/005 , A61K48/0091 , A61K2039/5158 , C12N7/00 , C12N15/86 , C12N15/8645 , C12N2750/14122 , C12N2750/14132 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14171 , C12N2810/6027
Abstract: Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.
Abstract translation: 公开了酪氨酸修饰的rAAV载体，以及包含它们的感染性病毒粒子，组合物和药物制剂。 还公开了制备方法和使用所公开的酪氨酸磷酸化衣壳蛋白突变体rAAV载体在多种诊断和治疗应用中的方法，包括体内和离体基因治疗以及大规模生产rAAV载体。
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公开(公告)号：US09920097B2
公开(公告)日：2018-03-20
申请号：US13899481
申请日：2013-05-21
Inventor： Li Zhong , Sergei Zolotukhin , Lakshmanan Govindasamy , Mavis Agbandje-McKenna , Arun Srivastava
IPC: C07K14/005 , C12N15/86 , A61K48/00
CPC classification number: C07K14/005 , A61K35/76 , A61K39/0011 , A61K48/0008 , A61K48/005 , A61K48/0091 , A61K2039/5158 , C12N7/00 , C12N15/86 , C12N15/8645 , C12N2750/14122 , C12N2750/14132 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14171 , C12N2810/6027
Abstract: Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using novel capsid-protein-mutated rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more symptoms of disease or abnormal conditions via in situ and/or ex vivo mammalian gene therapy methods. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.
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4.发明授权
公开(公告)号：US09157098B2
公开(公告)日：2015-10-13
申请号：US13855640
申请日：2013-04-02
Inventor： Li Zhong , Sergei Zolotukhin , Lakshmanan Govindasamy , Mavis Agbandje-McKenna , Arun Srivastava
IPC: C07K14/00 , C12N15/864 , A61K48/00 , C12N15/86
CPC classification number: C07K14/005 , A61K35/76 , A61K39/0011 , A61K48/0008 , A61K48/005 , A61K48/0091 , A61K2039/5158 , C12N7/00 , C12N15/86 , C12N15/8645 , C12N2750/14122 , C12N2750/14132 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14171 , C12N2810/6027
Abstract: Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.
Abstract translation: 公开了酪氨酸修饰的rAAV载体，以及包含它们的感染性病毒粒子，组合物和药物制剂。 还公开了制备方法和使用所公开的酪氨酸磷酸化衣壳蛋白突变体rAAV载体在多种诊断和治疗应用中的方法，包括体内和离体基因治疗以及大规模生产rAAV载体。
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5.发明申请
公开(公告)号：US20140341852A1
公开(公告)日：2014-11-20
申请号：US14214011
申请日：2014-03-14
Inventor： Arun Srivastava , George V. Aslanidi , Sergei Zolotukhin , Mavis Agbandje-McKenna , Kim M. Van Vliet , Li Zhong , Lakshmanan Govindasamy
IPC: C07K14/005 , C07K14/805 , A61K38/42 , C12N7/00 , C12N15/86
CPC classification number: C07K14/005 , A61K38/42 , C07K14/805 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2750/14171 , C12N2810/6027
Abstract: Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations containing them. Also provided are methods of preparing and using the disclosed capsid-protein-mutated rAAV constructs in a variety of diagnostic and therapeutic modalities, including, inter alia, as mammalian cell-targeting delivery agents, and as human gene therapy vectors. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.
Abstract translation: 公开了衣壳修饰的rAAV表达载体，以及感染性病毒粒子，组合物和含有它们的药物制剂。 还提供了制备和使用所公开的衣壳蛋白突变的rAAV构建体在各种诊断和治疗方式中的方法，所述方法尤其包括作为哺乳动物细胞靶向递送剂，以及作为人基因治疗载体。 还公开了用于衣壳修饰的rAAV表达载体，病毒颗粒和具有相对于未修饰的rAAV载体的转导效率具有改进的转导效率的感染性病毒粒子的大规模生产方法，以及在制造中使用所公开的组合物 的用于各种体外和/或体内应用的药物。
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公开(公告)号：US09775918B2
公开(公告)日：2017-10-03
申请号：US14847528
申请日：2015-09-08
Inventor： Li Zhong , Sergei Zolotukhin , Lakshmanan Govindasamy , Mavis Agbandje-McKenna , Arun Srivastava
IPC: A61K48/00 , C12N15/86 , C12N15/864 , A61K35/76 , C12N7/00
CPC classification number: C07K14/005 , A61K35/76 , A61K39/0011 , A61K48/0008 , A61K48/005 , A61K48/0091 , A61K2039/5158 , C12N7/00 , C12N15/86 , C12N15/8645 , C12N2750/14122 , C12N2750/14132 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14171 , C12N2810/6027
Abstract: Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.
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公开(公告)号：US20160106865A1
公开(公告)日：2016-04-21
申请号：US14847528
申请日：2015-09-08
Inventor： Li Zhong , Sergei Zolotukhin , Lakshmanan Govindasamy , Mavis Agbandje-McKenna , Arun Srivastava
CPC classification number: C07K14/005 , A61K35/76 , A61K39/0011 , A61K48/0008 , A61K48/005 , A61K48/0091 , A61K2039/5158 , C12N7/00 , C12N15/86 , C12N15/8645 , C12N2750/14122 , C12N2750/14132 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14171 , C12N2810/6027
Abstract: Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.
Abstract translation: 公开了酪氨酸修饰的rAAV载体，以及包含它们的感染性病毒粒子，组合物和药物制剂。 还公开了制备方法和使用所公开的酪氨酸磷酸化衣壳蛋白突变体rAAV载体在多种诊断和治疗应用中的方法，包括体内和离体基因治疗以及大规模生产rAAV载体。
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8.发明授权Therapeutic methods based on tyrosine-substituted, capsid-modified rAAV vectors 有权基于酪氨酸取代的衣壳修饰的rAAV载体的治疗方法
公开(公告)号：US08802440B2
公开(公告)日：2014-08-12
申请号：US13854011
申请日：2013-03-29
Inventor： Li Zhong , Sergei Zolotukhin , Lakshmanan Govindasamy , Mavis Agbandje-McKenna , Arun Srivastava
CPC classification number: C07K14/005 , A61K35/76 , A61K39/0011 , A61K48/0008 , A61K48/005 , A61K48/0091 , A61K2039/5158 , C12N7/00 , C12N15/86 , C12N15/8645 , C12N2750/14122 , C12N2750/14132 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14171 , C12N2810/6027
Abstract: Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.
Abstract translation: 公开了酪氨酸修饰的rAAV载体，以及包含它们的感染性病毒粒子，组合物和药物制剂。 还公开了制备方法和使用所公开的酪氨酸磷酸化衣壳蛋白突变体rAAV载体在多种诊断和治疗应用中的方法，包括体内和离体基因治疗以及大规模生产rAAV载体。
