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1. 发明申请

WO2005118792A1 COMPOSITIONS AND METHODS TO PREVENT AAV VECTOR AGGREGATION 审中-公开
标题翻译：组合物和方法，以防止AAV矢量聚集
公开(公告)号：WO2005118792A1
公开(公告)日：2005-12-15
申请号：PCT/US2005/019235
申请日：2005-06-01
申请人： AVIGEN, INC. , WRIGHT, John Fraser , QU, Guang
发明人： WRIGHT, John Fraser , QU, Guang
IPC分类号： C12N7/02
CPC分类号： C12N7/00 , C12N2750/14111 , C12N2750/14151
摘要： Compositions and methods are provided for preparation of concentrated stock solutions of AAV virions without aggregation. Formulations for AAV preparation and storage are high ionic strength solutions (e.g. µ ~500mM) that are nonetheless isotonic with the intended target tissue. This combination of high ionic strength and modest osmolarity is achieved using salts of high valency, such as sodium citrate. AAV stock solutions up to 6.4x1013 vg/mL are possible using the formulations of the invention, with no aggregation being observed even after ten freeze-thaw cycles. The surfactant Pluronic® F68 may be added at 0.001% to prevent losses of virions to surfaces during handling. Virion preparations can also be treated with nucleases to eliminate small nucleic acid strands on virions surfaces that exacerbate aggregation.
摘要翻译：提供组合物和方法用于制备没有聚集的AAV病毒体的浓缩储备溶液。用于AAV制备和储存的制剂是与目标组织等渗的高离子强度溶液（例如μ〜500mM）。高离子强度和适度渗透压的这种组合使用高价盐，例如柠檬酸钠。使用本发明的制剂可达到6.4×10 13 vg / mL的AAV储备溶液，即使在十次冻融循环后也没有观察到聚集。表面活性剂PluronicF68可以以0.001％添加以防止在处理期间损伤表面的病毒粒子。病毒颗粒制剂也可用核酸酶处理，以消除加重聚集的病毒粒子表面上的小核酸链。

2. 发明申请

WO2011094198A1 A SCALABLE MANUFACTURING PLATFORM FOR VIRAL VECTOR PURIFICATION AND VIRAL VECTORS SO PURIFIED FOR USE IN GENE THERAPY 审中-公开
标题翻译：用于病毒载体纯化的可扩展的制造平台和纯化用于基因治疗的病毒载体
公开(公告)号：WO2011094198A1
公开(公告)日：2011-08-04
申请号：PCT/US2011/022371
申请日：2011-01-25
申请人： THE CHILDREN'S HOSPITAL OF PHILADELPHIA RESEARCH INSTITUTE, ABRAMSON RESEARCH CENTER , WRIGHT, John, Fraser , QU, Guang , HAUCK, Bernd , HIGH, Katherine
发明人： WRIGHT, John, Fraser , QU, Guang , HAUCK, Bernd , HIGH, Katherine
IPC分类号： C12N15/86 , C12N7/02
CPC分类号： A61K39/23 , A61K38/465 , A61K48/00 , A61K48/0091 , C07K2/00 , C12N7/00 , C12N15/64 , C12N15/86 , C12N15/864 , C12N15/8645 , C12N2750/14011 , C12N2750/14051 , C12N2750/14111 , C12N2750/14143 , C12N2750/14151 , C12Y301/01064
摘要： Methods for preparing highly purified AAV vector formulations are provided. The highly pure AAV formulations described herein are superior for clinical use.
摘要翻译：提供了制备高纯度AAV载体制剂的方法。本文所述的高纯度AAV制剂优于临床应用。

3. 发明申请

WO2004113494A3 METHODS FOR PRODUCING PREPARATIONS OF RECOMBINANT AAV VIRIONS SUBSTANTIALLY FREE OF EMPTY CAPSIDS 审中-公开
公开(公告)号：WO2004113494A3
公开(公告)日：2004-12-29
申请号：PCT/US2004/015958
申请日：2004-05-21
申请人： AVIGEN, INC. , QU, Guang , WRIGHT, John Fraser
发明人： QU, Guang , WRIGHT, John Fraser
IPC分类号： C12N7/02
摘要： Methods for separating AAV empty capsids from mixtures of AAV vector particles and AAV empty capsids are described. The methods use column chromatography techniques and provide for commercially viable levels of recombinant AAV virions.

4. 发明申请

WO2004113494A2 METHODS FOR PRODUCING PREPARATIONS OF RECOMBINANT AAV VIRIONS SUBSTANTIALLY FREE OF EMPTY CAPSIDS 审中-公开
标题翻译：生产重组AAV病毒制剂的方法主要是无空白的CAPSIDS
公开(公告)号：WO2004113494A2
公开(公告)日：2004-12-29
申请号：PCT/US2004/015958
申请日：2004-05-21
申请人： AVIGEN, INC. , QU, Guang , WRIGHT, John Fraser
发明人： QU, Guang , WRIGHT, John Fraser
IPC分类号： C12N
CPC分类号： C12N15/86 , C12N7/00 , C12N2750/14143 , C12N2750/14151
摘要： Methods for separating AAV empty capsids from mixtures of AAV vector particles and AAV empty capsids are described. The methods use column chromatography techniques and provide for commercially viable levels of recombinant AAV virions.
摘要翻译：描述了从AAV载体颗粒和AAV空衣壳的混合物中分离AAV空衣壳的方法。该方法使用柱色谱技术并提供商业上可行的重组AAV病毒粒子水平。

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