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    OPTIMIZED FACTOR IX GENE
    94.
    发明申请
    OPTIMIZED FACTOR IX GENE 有权

    公开(公告)号：US20250154489A1

    公开(公告)日：2025-05-15

    申请号：US19029664

    申请日：2025-01-17

    Applicant: Bioverativ Therapeutics Inc.

    Inventor： Siyuan TAN ,  Robert T. PETERS ,  Tongyao LIU

    IPC: C12N9/64

    Abstract: The present invention provides codon optimized Factor IX sequences, vectors and host cells comprising codon optimized Factor IX sequences, polypeptides encoded by codon optimized Factor IX sequences, and methods of producing such polypeptides. The present invention also provides methods of treating bleeding disorders such as hemophilia comprising administering to the subject a codon optimized Factor IX nucleic acid sequence or the polypeptide encoded thereby.

    Optimized factor IX gene
    96.
    发明授权
    Optimized factor IX gene 有权

    公开(公告)号：US12275970B2

    公开(公告)日：2025-04-15

    申请号：US17060759

    申请日：2020-10-01

    Applicant: Bioverativ Therapeutics Inc.

    Inventor： Siyuan Tan ,  Robert T. Peters ,  Tongyao Liu

    IPC: C12N9/64

    Abstract: The present invention provides codon optimized Factor IX sequences, vectors and host cells comprising codon optimized Factor IX sequences, polypeptides encoded by codon optimized Factor IX sequences, and methods of producing such polypeptides. The present invention also provides methods of treating bleeding disorders such as hemophilia comprising administering to the subject a codon optimized Factor IX nucleic acid sequence or the polypeptide encoded thereby.

    Nucleic acid molecules and uses thereof
    97.
    发明授权
    Nucleic acid molecules and uses thereof 有权

    公开(公告)号：US12168776B2

    公开(公告)日：2024-12-17

    申请号：US16636583

    申请日：2018-08-09

    Applicant: BIOVERATIV THERAPEUTICS INC.

    Inventor： Alexey Seregin ,  Tongyao Liu ,  Susannah Patarroyo-White ,  Douglas Drager ,  Robert T. Peters ,  Jiayun Liu

    IPC: C12N15/00 ,  C07K14/755 ,  C12N15/86

    Abstract: The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a miRNA and/or a therapeutic protein. In certain embodiments, the therapeutic protein comprises a clotting factor, e.g., a FVIII polypeptide, a FIX polypeptide, or a fragment thereof. In some embodiments, the first ITR and/or the second ITR is an ITR of a non-adeno-associated virus (AAV). The present disclosure also provides methods of treating bleeding disorders such as hemophilia comprising administering to the subject the nucleic acid molecule or a polypeptide encoded thereby.

    BLOOD FACTOR MONITORING ASSAY AND USES THEREOF
    99.
    发明申请
    BLOOD FACTOR MONITORING ASSAY AND USES THEREOF 有权

    公开(公告)号：US20220412996A1

    公开(公告)日：2022-12-29

    申请号：US17749834

    申请日：2022-05-20

    Applicant: Bioverativ Therapeutics Inc.

    Inventor： Jurg SOMMER

    IPC: G01N33/86 ,  G01N33/92 ,  A61K38/48

    Abstract: The present disclosure provides methods and compositions for diagnosing and treating subject having a bleeding disorder. The disclosed methods comprise contacting a sample, e.g., a blood or plasma sample obtained from the patient, with an activation mixture comprising an activated coagulation factor and a phospholipid mixture, wherein the activation mixture is dried onto a solid substrate. Also provided is a global hemostasis test based on the integration of clotting time (Ct) and pharmacokinetics data. The methods and compositions presented can be applied to point-of-care diagnostic systems.

    FACTOR VIII CHIMERIC PROTEINS AND USES THEREOF
    100.
    发明申请
    FACTOR VIII CHIMERIC PROTEINS AND USES THEREOF 有权

    公开(公告)号：US20220275057A1

    公开(公告)日：2022-09-01

    申请号：US17519719

    申请日：2021-11-05

    Applicant: Bioverativ Therapeutics Inc.

    Inventor： Ekta Seth CHHABRA ,  Tongyao LIU ,  Robert T. PETERS ,  John KULMAN

    IPC: C07K14/755 ,  C07K16/00

    Abstract: The present invention provides a chimeric protein comprising a first polypeptide which comprises a FVIII protein and a first Ig constant region or a portion thereof and a second polypeptide which comprises a VWF protein comprising the D′ domain and D3 domain of VWF, a XTEN sequence having less than 288 amino acids in length, and a second Ig constant region or a portion thereof, wherein the first polypeptide and the second polypeptide are associated with each other. The invention also includes nucleotides, vectors, host cells, methods of using the chimeric proteins.

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