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    Scalable production method for AAV
    1.
    发明授权
    Scalable production method for AAV 有权
    AAV可扩展生产方法

    公开(公告)号：US09198984B2

    公开(公告)日：2015-12-01

    申请号：US12226588

    申请日：2007-04-27

    申请人： Martin Lock ,  Luc H Vandenberghe ,  James M. Wilson

    发明人： Martin Lock ,  Luc H Vandenberghe ,  James M. Wilson

    IPC分类号： A61K38/00 ,  C12N7/00 ,  A61K48/00 ,  C07K14/005

    CPC分类号： C12N7/00 ,  A61K48/0091 ,  C07K14/005 ,  C12N2750/14151

    摘要： A method for producing AAV, without requiring cell lysis, is described. The method involves harvesting AAV from the supernatant. For AAV having capsids with a heparin binding site, the method involves modifying the AAV capsids and/or the culture conditions to ablate the binding between the AAV heparin binding site and the cells, thereby allowing the AAV to pass into the supernatant, i.e., media. Thus, the method of the invention provides supernatant containing high yields of AAV which have a higher degree of purity from cell membranes and intracellular materials, as compared to AAV produced using methods using a cell lysis step.

    摘要翻译： 描述了不需要细胞裂解的生产AAV的方法。 该方法包括从上清液中收获AAV。 对于具有肝素结合位点的衣壳的AAV，该方法包括修饰AAV衣壳和/或培养条件以消除AAV肝素结合位点与细胞之间的结合，从而允许AAV进入上清液，即培养基 。 因此，与使用细胞裂解步骤的方法产生的AAV相比，本发明的方法提供了含有高产率的AAV的上清液，其具有比细胞膜和细胞内物质更高的纯度。

    Scalable Production Method for AAV
    2.
    发明申请
    Scalable Production Method for AAV 有权
    AAV的可扩展生产方法

    公开(公告)号：US20090275107A1

    公开(公告)日：2009-11-05

    申请号：US12226588

    申请日：2007-04-27

    申请人： Martin Lock ,  Luc H. Vandenberghe ,  James M. Wilson

    发明人： Martin Lock ,  Luc H. Vandenberghe ,  James M. Wilson

    IPC分类号： C12N7/02 ,  C12N7/00

    CPC分类号： C12N7/00 ,  A61K48/0091 ,  C07K14/005 ,  C12N2750/14151

    摘要： A method for producing AAV, without requiring cell lysis, is described. The method involves harvesting AAV from the supernatant. For AAV having capsids with a heparin binding site, the method involves modifying the AAV capsids and/or the culture conditions to ablate the binding between the AAV heparin binding site and the cells, thereby allowing the AAV to pass into the supernatant, i.e., media. Thus, the method of the invention provides supernatant containing high yields of AAV which have a higher degree of purity from cell membranes and intracellular materials, as compared to AAV produced using methods using a cell lysis step.

    摘要翻译： 描述了不需要细胞裂解的生产AAV的方法。 该方法包括从上清液中收获AAV。 对于具有肝素结合位点的衣壳的AAV，该方法包括修饰AAV衣壳和/或培养条件以消除AAV肝素结合位点与细胞之间的结合，从而允许AAV进入上清液，即培养基 。 因此，与使用细胞裂解步骤的方法产生的AAV相比，本发明的方法提供了含有高产率的AAV的上清液，其具有比细胞膜和细胞内物质更高的纯度。

    Compositions and Methods for Altering Tissue Specificity and Improving AAV9-Mediated Gene Transfer
    8.
    发明申请
    Compositions and Methods for Altering Tissue Specificity and Improving AAV9-Mediated Gene Transfer 有权
    改变组织特异性和改善AAV9介导的基因转移的组合物和方法

    公开(公告)号：US20130323226A1

    公开(公告)日：2013-12-05

    申请号：US13985630

    申请日：2012-02-17

    申请人： James M. Wilson ,  Christie L. Bell ,  Luc H. Vandenberghe

    发明人： James M. Wilson ,  Christie L. Bell ,  Luc H. Vandenberghe

    IPC分类号： A61K31/7088 ,  A61K31/685 ,  A61K38/47

    CPC分类号： A61K31/7088 ,  A61K31/685 ,  A61K38/47 ,  C12N15/86 ,  C12N2750/14043 ,  C12N2750/14045 ,  C12N2810/6027

    摘要： A method of altering the targeting and/or cellular uptake efficiency of an adeno-associated virus (AAV) viral vector having a capsid containing an AAV9 cell surface binding domain is described. The method involves modifying a clade F cell surface receptor which comprises a glycan having a terminal sialic acid residue and a penultimate β-galactose residue. The modification may involve retargeting the vector by temporarily functionally ablate AAV9 binding in a subset of cells, thereby redirecting the vector to another subset of cells. Alternatively, the modification may involve increasing cellular update efficiency by treating the cells with a neuraminidase to expose cell surface β-galactose. Also provided are compositions containing the AAV9 vector and a neuraminidase. Also provided is a method for purifying AAV9 using β-galactose linked to solid support. Also provided are mutant vectors which have been modified to alter their targeting specificity, including mutant AAV9 in which the galactose binding domain is mutated and AAV in which an AAV9 galactose binding domain is engineered.

    摘要翻译： 描述了改变具有包含AAV9细胞表面结合结构域的衣壳的腺相关病毒（AAV）病毒载体的靶向和/或细胞摄取效率的方法。 该方法包括修饰进化枝F细胞表面受体，其包含具有末端唾液酸残基的聚糖和倒数第二半乳糖残基。 该修饰可能涉及通过临时功能地消融细胞子集中的AAV9结合来重新定向载体，从而将载体重定向到另一个细胞亚群。 或者，修饰可以涉及通过用神经氨酸酶处理细胞以暴露细胞表面β-半乳糖来增加细胞更新效率。 还提供了含有AAV9载体和神经氨酸酶的组合物。 还提供了使用与固体支持物连接的β-半乳糖来纯化AAV9的方法。 还提供已被修饰以改变其靶向特异性的突变载体，包括其中半乳糖结合结构域突变的突变AAV9和其中AAV9半乳糖结合结构域被工程化的AAV。

    Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
    10.
    发明申请
    Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof 审中-公开
    具有降低衣壳免疫原性的改良的AAV载体及其用途

    公开(公告)号：US20090317417A1

    公开(公告)日：2009-12-24

    申请号：US12226536

    申请日：2007-04-27

    申请人： Luc H. Vandenberghe ,  James M. Wilson

    发明人： Luc H. Vandenberghe ,  James M. Wilson

    IPC分类号： A61K39/235 ,  C12N7/00 ,  A61P31/12

    CPC分类号： C12N15/86 ,  A61K2039/57 ,  C12N2750/14142 ,  C12N2750/14143

    摘要： A method of reducing the cellular immune response and/or toxicity of AAV-mediated delivery is described. The method provides for masking or ablating a RxxR motif which induces T-cells, and which is located on select AAV capsids. The method further provides for reducing or eliminating heparin binding to an AAV. Also provided are compositions containing modified AAV capsids and methods of using same.

    摘要翻译： 描述了减少AAV介导的递送的细胞免疫应答和/或毒性的方法。 该方法提供掩蔽或消融诱导T细胞的RxxR基序，其位于选定的AAV衣壳上。 该方法进一步提供了减少或消除肝素与AAV的结合。 还提供了含有修饰的AAV衣壳的组合物及其使用方法。