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    ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF
    1.
    发明申请
    ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF 有权

    公开(公告)号：US20210380643A1

    公开(公告)日：2021-12-09

    申请号：US17400041

    申请日：2021-08-11

    Applicant: 4D MOLECULAR THERAPEUTICS INC.

    Inventor： David H. KIRN ,  Melissa KOTTERMAN ,  David SCHAFFER

    IPC: C07K14/005 ,  C12N7/00 ,  A61P21/00 ,  A61K9/00 ,  A61K38/47

    Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of muscle cells as compared to the infectivity of the muscle cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more muscle cells for the treatment of muscle disorders and diseases.

    ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF
    2.
    发明申请
    ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF 有权

    公开(公告)号：US20210069348A1

    公开(公告)日：2021-03-11

    申请号：US16951984

    申请日：2020-11-18

    Applicant: 4D MOLECULAR THERAPEUTICS INC.

    Inventor： David H. KIRN ,  Melissa KOTTERMAN ,  David SCHAFFER

    IPC: A61K48/00 ,  A61P27/02 ,  A61K9/00 ,  C07K14/005 ,  C12N15/86

    Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.

    ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND USE FOR INHIBITING ANGIOGENESIS
    3.
    发明申请
    ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND USE FOR INHIBITING ANGIOGENESIS 审中-公开

    公开(公告)号：US20200282077A1

    公开(公告)日：2020-09-10

    申请号：US16765758

    申请日：2018-11-26

    Applicant: 4D MOLECULAR THERAPEUTICS INC.

    Inventor： David H. KIRN ,  Melissa KOTTERMAN ,  David SCHAFFER ,  Paul SZYMANSKI ,  Peter FRANCIS

    IPC: A61K48/00 ,  C12N7/00 ,  C07K14/005 ,  A61K9/00 ,  C07K16/22

    Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.

    ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF
    6.
    发明申请
    ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF 审中-公开

    公开(公告)号：US20190255192A1

    公开(公告)日：2019-08-22

    申请号：US16300446

    申请日：2017-05-12

    Applicant: 4D MOLECULAR THERAPEUTICS INC.

    Inventor： David H. KIRN ,  Melissa KOTTERMAN ,  David SCHAFFER

    IPC: A61K48/00 ,  C12N15/86 ,  C07K14/005 ,  A61P27/02 ,  A61K9/00

    Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AA V virion comprising the unmodified parental capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.

    TREATMENTS FOR RETINAL DISEASE
    7.
    发明申请
    TREATMENTS FOR RETINAL DISEASE 有权

    公开(公告)号：US20230001018A1

    公开(公告)日：2023-01-05

    申请号：US17746443

    申请日：2022-05-17

    Applicant: 4D MOLECULAR THERAPEUTICS INC.

    Inventor： David H. KIRN ,  Melissa KOTTERMAN ,  David SCHAFFER

    IPC: A61K48/00 ,  A61K9/00 ,  A61P27/02 ,  C07K14/005 ,  C12N15/86

    Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more ceils of the retina for the treatment of retinal disorders and diseases.

    ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF
    8.
    发明申请
    ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF 有权

    公开(公告)号：US20220251145A1

    公开(公告)日：2022-08-11

    申请号：US17718870

    申请日：2022-04-12

    Applicant: 4D MOLECULAR THERAPEUTICS INC.

    Inventor： David H. KIRN ,  Melissa KOTTERMAN ,  David SCHAFFER

    IPC: C07K14/005 ,  A61K38/47 ,  A61K38/44 ,  C12N9/10 ,  C12N15/86 ,  A61K38/17

    Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of muscle cells as compared to the infectivity of the muscle cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more muscle cells for the treatment of muscle disorders and diseases.

    ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF
    10.
    发明申请
    ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF 有权

    公开(公告)号：US20210162071A1

    公开(公告)日：2021-06-03

    申请号：US17163093

    申请日：2021-01-29

    Applicant: 4D MOLECULAR THERAPEUTICS INC.

    Inventor： David H. KIRN ,  Melissa KOTTERMAN ,  David SCHAFFER

    IPC: A61K48/00 ,  A61P27/02 ,  C07K14/005 ,  A61K9/00 ,  C12N15/86

    Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.

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