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    AAV-MEDIATED DELIVERY OF THERAPEUTIC ANTIBODIES TO THE INNER EAR
    2.
    发明申请
    AAV-MEDIATED DELIVERY OF THERAPEUTIC ANTIBODIES TO THE INNER EAR 有权

    公开(公告)号：US20210363499A1

    公开(公告)日：2021-11-25

    申请号：US17392910

    申请日：2021-08-03

    申请人： Akouos, Inc.

    发明人： Emmanuel John Simons ,  Robert Ng ,  Michael McKenna

    IPC分类号： C12N7/00 ,  A61K9/00 ,  A61K48/00 ,  C07K16/22

    摘要： Provided herein are methods that include introducing into an inner ear of a mammal a therapeutically effective amount of an adeno-associated virus (AAV) vector that includes a nucleotide sequence encoding (a) a polypeptide including an antibody heavy chain variable domain operably linked to a signal peptide and a polypeptide including an antibody light chain variable domain operably linked to a signal peptide; (b) a polypeptide including an antigen-binding antibody fragment operably linked to a signal peptide; or (c) a soluble vascular endothelial growth factor receptor operably linked to a signal peptide.

    USE OF ADENO-ASSOCIATED VIRAL VECTORS TO CORRECT GENE DEFECTS/ EXPRESS PROTEINS IN HAIR CELLS AND SUPPORTING CELLS IN THE INNER EAR
    8.
    发明申请
    USE OF ADENO-ASSOCIATED VIRAL VECTORS TO CORRECT GENE DEFECTS/ EXPRESS PROTEINS IN HAIR CELLS AND SUPPORTING CELLS IN THE INNER EAR 有权

    公开(公告)号：US20230056182A1

    公开(公告)日：2023-02-23

    申请号：US17291931

    申请日：2019-11-07

    申请人： Akouos, Inc.

    发明人： Emmanuel John Simons ,  Robert Ng ,  Danielle R. Lenz ,  Michelle Valero

    IPC分类号： A61K48/00 ,  A61P27/16 ,  C12N15/86

    摘要： Provided herein are compositions that include at least two different nucleic acid vectors that, when introduced into a primate cell, the at least two different vectors undergo con catamerization or homologous recombination with each other, thereby forming a recombined nucleic acid that encodes a full-length target protein (e.g., a supporting cell target protein or a hair cell target protein). Also provided are compositions that include a single AAV vector that, when introduced into a primate cell, a nucleic acid encoding a full-length target protein (e.g., a supporting cell target protein or a hair cell target protein) is generated at the locus of the supporting cell target gene, and the primate expresses the target protein (e.g., a supporting cell target protein or a hair cell target protein).