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    SCALABLE PRODUCTION METHOD FOR AAV
    1.
    发明申请
    SCALABLE PRODUCTION METHOD FOR AAV 有权

    公开(公告)号：US20230116968A1

    公开(公告)日：2023-04-20

    申请号：US17806005

    申请日：2022-06-08

    申请人： The Trustees of the University of Pennsylvania

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

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

    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.

    Scalable production method for AAV
    2.
    发明授权
    Scalable production method for AAV 有权

    公开(公告)号：US11377641B2

    公开(公告)日：2022-07-05

    申请号：US16998548

    申请日：2020-08-20

    申请人： The Trustees of the University of Pennsylvania

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

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

    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.

    Compositions and Methods for Altering Tissue Specificity and Improving AAV9-Mediated Gene Transfer
    9.
    发明申请
    Compositions and Methods for Altering Tissue Specificity and Improving AAV9-Mediated Gene Transfer 有权

    公开(公告)号：US20210145857A1

    公开(公告)日：2021-05-20

    申请号：US17159735

    申请日：2021-01-27

    申请人： The Trustees of The University of Pennsylvania

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

    IPC分类号： A61K31/7088 ,  C12N15/86 ,  A61K31/685 ,  A61K38/47

    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.