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    CHIMERIC FACTOR VIII POLYPEPTIDES AND USES THEREOF
    1.
    发明申请
    CHIMERIC FACTOR VIII POLYPEPTIDES AND USES THEREOF 有权

    公开(公告)号：US20230011438A1

    公开(公告)日：2023-01-12

    申请号：US17826932

    申请日：2022-05-27

    Applicant: BIOVERATIV THERAPEUTICS INC.

    Inventor： Ekta Seth CHHABRA ,  Tongyao LIU ,  Robert T. PETERS ,  Haiyan JIANG

    IPC: C07K14/755 ,  A61K47/62 ,  C07K16/00

    Abstract: The present invention provides a VWF fragment comprising the D′ domain and D3 domain of VWF, a chimeric protein comprising the VWF fragment and a heterologous moiety, or a chimeric protein comprising the VWF fragment and a FVIII protein and methods of using the same. A polypeptide chain comprising a VWF fragment of the invention binds to or is associated with a polypeptide chain comprising a FVIII protein and the polypeptide chain comprising the VWF fragment can prevent or inhibit binding of endogenous VWF to the FVIII protein. By preventing or inhibiting binding of endogenous VWF to the FVIII, which is a half-life limiting factor for FVIII, the VWF fragment can induce extension of half-life of the FVIII protein. The invention also includes nucleotides, vectors, host cells, methods of using the VWF fragment, or the chimeric proteins.

    USE OF LENTIVIRAL VECTORS EXPRESSING FACTOR VIII
    3.
    发明申请
    USE OF LENTIVIRAL VECTORS EXPRESSING FACTOR VIII 有权

    公开(公告)号：US20210038744A1

    公开(公告)日：2021-02-11

    申请号：US16965895

    申请日：2019-01-31

    Applicant: BIOVERATIV THERAPEUTICS INC.

    Inventor： Andrea ANNONI ,  Alessio CANTORE ,  Douglas DRAGER ,  Tongyao LIU ,  Michela MILANI ,  Jeff MOFFIT ,  Luigi NALDINI ,  Susannah PATARROYO-WHITE ,  Robert T. PETERS ,  Alexey SEREGIN

    IPC: A61K48/00 ,  A61K38/37

    Abstract: The present disclosure provides lentiviral vectors comprising codon optimized Factor VIII sequences, and methods of using such lentiviral vectors. The liver-targeted lentiviral vectors disclosed herein can be used for gene therapy, wherein the lentiviral gene delivery enables stable integration of the transgene expression cassette into the genome of targeted cells (e.g., hepatocytes) of pediatric (e.g., neonatal) or adult subjects, achieving an improvement in FVIII expression (for example, a 100-fold improvement) at low lentiviral vector doses (e.g., 5×1010 or lower, such as 1.5×109 or lower, or 1×108 TU/kg or lower). The present disclosure also provides methods of treating bleeding disorders such as hemophilia (e.g., hemophilia A) comprising administering to a subject in need thereof a liver-targeted lentiviral vector comprising a codon optimized Factor VIII nucleic acid sequence at low dosages (1×108 TU/kg or lower to 1.5×1010 TU/kg).

    THROMBIN CLEAVABLE LINKER WITH XTEN AND ITS USES THEREOF
    4.
    发明公开
    THROMBIN CLEAVABLE LINKER WITH XTEN AND ITS USES THEREOF 审中-公开

    公开(公告)号：US20240083975A1

    公开(公告)日：2024-03-14

    申请号：US18358601

    申请日：2023-07-25

    Applicant: Bioverativ Therapeutics Inc.

    Inventor： Ekta Seth CHHABRA ,  John KULMAN ,  Tongyao LIU

    IPC: C07K14/755 ,  A61K38/37

    CPC classification number: C07K14/755 ,  A61K38/37 ,  A61K38/00

    Abstract: The present invention provides a chimeric molecule comprising a VWF protein fused to a heterologous moiety via a VWF linker. The invention provides an efficient VWF linker that can be cleaved in the presence of thrombin. The chimeric molecule can further comprise a polypeptide chain comprising a FVIII protein and a second heterologous moiety, wherein the chain comprising the VWF protein and the chain comprising the FVIII protein are associated with each other. The invention also includes nucleotides, vectors, host cells, methods of using the chimeric proteins.

    OPTIMIZED FACTOR IX GENE
    6.
    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.

    FACTOR VIII CHIMERIC PROTEINS AND USES THEREOF
    7.
    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.

    NUCLEIC ACID MOLECULES AND USES THEREOF
    8.
    发明申请
    NUCLEIC ACID MOLECULES AND USES THEREOF 有权

    公开(公告)号：US20210163986A1

    公开(公告)日：2021-06-03

    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: C12N15/86 ,  C07K14/755

    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.

    FACTOR VIII COMPLEX WITH XTEN AND VON WILLEBRAND FACTOR PROTEIN, AND USES THEREOF
    10.
    发明申请
    FACTOR VIII COMPLEX WITH XTEN AND VON WILLEBRAND FACTOR PROTEIN, AND USES THEREOF 审中-公开

    公开(公告)号：US20190169267A1

    公开(公告)日：2019-06-06

    申请号：US16154310

    申请日：2018-10-08

    Applicant: Bioverativ Therapeutics Inc.

    Inventor： Ekta Seth CHHABRA ,  Tongyao LIU ,  Pei-yun CHANG ,  Robert T. PETERS ,  John KULMAN

    IPC: C07K14/755 ,  A61K47/55 ,  C12N15/11 ,  C07K16/00 ,  A61K38/36 ,  A61K38/37

    Abstract: The present invention provides a chimeric protein comprising a VWF protein comprising the D′ domain and D3 domain of VWF, one or more XTEN sequence, and a FVIII protein, wherein the VWF fragment, the XTEN sequence, or the FVIII protein are linked to or associated with each other. The chimeric protein can further comprise one or more Ig constant region or a portion thereof (e.g., an Fc region). A polypeptide chain comprising a VWF fragment of the invention binds to or is associated with a polypeptide chain comprising a FVIII protein linked to an XTEN sequence and the polypeptide chain comprising the VWF fragment can prevent or inhibit binding of endogenous VWF to the FVIII protein linked to the XTEN sequence. By preventing or inhibiting binding of endogenous VWF to the FVIII protein, which is a half-life limiting factor for FVIII, the VWF fragment can induce extension of half-life of the chimeric protein comprising a FVIII protein. The invention also includes nucleotides, vectors, host cells, methods of using the VWF fragment, or the chimeric proteins.

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