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    COMPOSITIONS COMPRISING AAV EXPRESSING DUAL ANTIBODY CONSTRUCTS AND USES THEREOF
    41.
    发明公开
    COMPOSITIONS COMPRISING AAV EXPRESSING DUAL ANTIBODY CONSTRUCTS AND USES THEREOF 审中-公开
    中两种抗体构建和组合物AAV表达及其用途

    公开(公告)号：EP3142750A1

    公开(公告)日：2017-03-22

    申请号：EP15792528.0

    申请日：2015-05-13

    申请人： The Trustees of The University of Pennsylvania

    发明人： WILSON, James, M. ,  TRETIAKOVA, Anna

    IPC分类号： A61P31/12 ,  C12N15/861

    CPC分类号： C07K16/1018 ,  A61K48/0008 ,  A61K2039/507 ,  C07K2317/14 ,  C07K2317/31 ,  C07K2317/51 ,  C07K2317/515 ,  C07K2317/52 ,  C07K2317/622 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14143 ,  C12N2830/205 ,  C12N2830/30 ,  C12N2830/50 ,  C12N2840/203

    摘要： A recombinant adeno-associated virus (AAV) having an AAV capsid and packaged therein a heterologous nucleic acid which expresses two functional antibody constructs in a cell is described. Also described are antibodies comprising a heavy chain and a light chain from a heterologous antibody. In one embodiment, the antibodies are co-expressed from a vector containing: a first expression cassette which encodes at least a first open reading frame (ORF) for a first immunoglobulin under the control of regulatory control sequences which direct expression thereof; and a second expression cassette which comprises a second ORF, a linker, and a third ORF under the control of regulatory control sequences which direct expression thereof, wherein the second and third ORF for a second and third immunoglobulin construct. The vector co-expressing these two antibody constructs is in one embodiment an AAV, in which the 5 ' and 3 ' ITRs flank the expression cassettes and regulatory sequences.

    摘要翻译： 具有一种重组腺相关病毒（AAV）AAV衣壳到及所包装的异源核酸表达列车在细胞中两个功能性抗体构建体进行说明。 如此描述的抗体包含来自异抗体重链和下车链。 在一个中，实施方式的抗体从包含矢量共同过表达：第一表达盒，其中至少用于调控序列的控制的其直接表达下的第一免疫球蛋白编码第一开放阅读框（ORF）; 和第二表达盒哪家直接表达它们，worin第二和第三ORF编码第二和第三免疫球蛋白构建体的调控序列的控制下，包括第二ORF，左，和第三ORF。 载体共表达本文两条抗体构建体是在AAV中的一个实施例，其中的5“和3”侧翼的ITR的表达盒和调节序列。

    METHODS AND COMPOSITIONS FOR TREATING METASTATIC BREAST CANCER AND OTHER CANCERS IN THE BRAIN
    42.
    发明公开
    METHODS AND COMPOSITIONS FOR TREATING METASTATIC BREAST CANCER AND OTHER CANCERS IN THE BRAIN 审中-公开
    方法和组合物用于治疗转移性乳腺癌和其他癌症在脑神经

    公开(公告)号：EP3134115A1

    公开(公告)日：2017-03-01

    申请号：EP15782834.4

    申请日：2015-04-24

    申请人： The Trustees of The University of Pennsylvania

    发明人： WILSON, James, M. ,  ROTHWELL, William, Thomas ,  HINDERER, Christian

    IPC分类号： A61K39/00 ,  A61K39/395 ,  C12N15/00 ,  C12N15/86 ,  C07H21/00

    CPC分类号： A61K48/005 ,  A61K9/0085 ,  C07K16/32 ,  C07K2317/24 ,  C12N2750/14143

    摘要： A composition comprising at least one AAV vector formulated for central nervous system delivery is described. The composition comprises at least one expression cassette which contains sequences encoding an anti-neoplastic immunoglobulin construct for delivery to the brain operably linked to expression control sequences therefor and a pharmaceutically acceptable carrier. The anti-neoplastic immunoglobulin construct may be an immunoglobulin modified to have decreased or no measurable affinity for neonatal Fc receptor (FcRn). Also provided are methods of using these constructs in preparing pharmaceutical compositions and uses thereof in anti-neoplastic regimens, particularly for primary and/or metastatic cancers of the brain.

    摘要翻译： 包含被配制用于中枢神经系统递送至少一个AAV载体的组合物进行说明。 所述组合物包含至少一个表达盒里面包含抗肿瘤免疫球蛋白序列编码构建用于传递到可操作地连接到为此表达控制序列和药学上可接受的载体中的脑。 抗肿瘤免疫球蛋白构建体可以被修饰以具有降低的免疫球蛋白或新生儿Fc受体（FcRn）没有可测量的亲和力的。 如此提供的是在制备药物组合物使用这些构建体的方法和在抗肿瘤治疗方案及其用途，特别是用于脑的初级和/或转移性的癌症。

    COMPOSITIONS AND METHODS FOR TREATING MPSI
    44.
    发明公开
    COMPOSITIONS AND METHODS FOR TREATING MPSI 审中-公开
    组合物以及治疗方法MPSI

    公开(公告)号：EP2984166A1

    公开(公告)日：2016-02-17

    申请号：EP14770186.6

    申请日：2014-03-13

    申请人： The Trustees Of The University Of Pennsylvania

    发明人： WILSON, James, M. ,  GURDA, Brittney, L.

    IPC分类号： C12N7/00 ,  C12N7/01 ,  C12N15/00

    摘要： A vector having an expression cassette having a hIDUA gene having a sequence of SEQ ID NO: 1 or a sequence at least about 95% identical thereto which encodes a functional human alpha-L-iduronidase is provided. The vector may be production vector or a rAAV8. Also provided are compositions containing these vectors and methods of treating MPSI and the symptoms associated with Hurler, Hurle-Scheie and Scheie syndromes.

    PHARMACOLOGICALLY INDUCED TRANSGENE ABLATION SYSTEM
    48.
    发明公开
    PHARMACOLOGICALLY INDUCED TRANSGENE ABLATION SYSTEM 审中-公开
    系统化学药理学杂志TRANSGENE ABRATION

    公开(公告)号：EP2553106A2

    公开(公告)日：2013-02-06

    申请号：EP11713419.7

    申请日：2011-03-28

    申请人： The Trustees Of The University Of Pennsylvania

    发明人： WILSON, James, M. ,  CHEN, Shu-Jen ,  TRETIAKOVA, Anna, P.

    IPC分类号： C12N15/86 ,  C12N15/864 ,  A61K48/00

    CPC分类号： C12N15/86 ,  A61K48/0066 ,  C07K2319/715 ,  C07K2319/80 ,  C12N9/22 ,  C12N2750/14143 ,  C12N2800/30 ,  C12N2800/80 ,  C12N2830/002 ,  C12N2830/003 ,  C12N2830/005 ,  C12N2830/006 ,  C12N2830/20 ,  C12N2840/203

    摘要： The present invention relates to gene therapy systems designed for the delivery of a therapeutic product to a subject using replication-defective virus composition(s) engineered with a built-in safety mechanism for ablating the therapeutic gene product, either permanently or temporarily, in response to a pharmacological agent - preferably an oral formulation, e.g., a pill. The invention is based, in part, on the applicants' development of an integrated approach, referred to herein as "PITA" (Pharmacologically Induced Transgene Ablation), for ablating a transgene or negatively regulating transgene expression. In this approach, replication-deficient viruses are used to deliver a transgene encoding a therapeutic product (an RNA or a protein) so that it is expressed in the subject, but can be reversibly or irreversibly turned off by administering the pharmacological agent; e.g., by administration of a small molecule that induces expression of an ablator specific for the transgene or its RNA transcript.

    摘要翻译： 本发明涉及基因治疗系统，其设计用于使用使用内置安全机制工程化的复制缺陷病毒组合物将治疗产品递送至受试者，所述安全机制永久地或临时地用于消融治疗性基因产物 药物剂 - 优选口服制剂，例如丸剂。 本发明部分地基于申请人开发用于消融转基因或负调节转基因表达的综合方法（本文称为PITA（Pharmacological Induced Transgene Ablation））。 在这种方法中，使用复制缺陷病毒递送编码治疗产物（RNA或蛋白质）的转基因，使其在受试者中表达，但可以通过施用药理学试剂来逆转或不可逆地关闭; 例如通过施用诱导特异于转基因或其RNA转录物的消融体表达的小分子。