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    METHOD OF SEPARATING FACTOR VIII FROM BLOOD PRODUCTS
    3.
    发明申请
    METHOD OF SEPARATING FACTOR VIII FROM BLOOD PRODUCTS 审中-公开
    从血液制品中分离因子VIII的方法

    公开(公告)号：WO2017137583A1

    公开(公告)日：2017-08-17

    申请号：PCT/EP2017/053045

    申请日：2017-02-10

    Applicant: OCTAPHARMA AG

    Inventor： WINGE, Stefan ,  ROSÉN, Per ,  SCHEEPERS, Alex

    IPC: C07K14/755 ,  B01D15/38 ,  C07K16/36

    CPC classification number: C07K14/755 ,  B01D15/3809 ,  C07K16/36

    Abstract: The invention provides a method for separating a Factor VIII (FVIII) protein from a first composition comprising the FVIII protein, which contains at least the light chain of FVIII, and a von-Willebrand-Factor (vWF) protein which comprises at least the FVIII binding domain of vWF, wherein the FVIII protein can form a complex with the vWF protein, the method comprising the steps: contacting the first composition with an affinity resin comprising a ligand and a matrix, wherein the ligand has an affinity to the light chain of FVIII, and separating the affinity resin from the mixture to obtain a modified first composition and a second composition, wherein the second composition contains the affinity resin, and a complex of the FVIII protein and the vWF protein.

    Abstract translation: 本发明提供了从包含FVIII蛋白的第一组合物中分离因子VIII（FVIII）蛋白的方法，所述FVIII蛋白至少含有FVIII的轻链和von-Willebrand因子（vWF ）蛋白，其至少包含vWF的FVIII结合结构域，其中所述FVIII蛋白可与vWF蛋白形成复合物，所述方法包括以下步骤：使所述第一组合物与包含配体和基质的亲和树脂接触，其中所述配体 对FVIII的轻链具有亲和力，并将亲和树脂与混合物分离以获得修饰的第一组合物和第二组合物，其中第二组合物含有亲和树脂以及FVIII蛋白和vWF蛋白的复合物。

    MUTATED TRUNCATED VON WILLEBRAND FACTOR
    4.
    发明申请
    MUTATED TRUNCATED VON WILLEBRAND FACTOR 审中-公开
    突变的VON WILLEBRAND因子

    公开(公告)号：WO2017117631A1

    公开(公告)日：2017-07-13

    申请号：PCT/AU2017/050010

    申请日：2017-01-06

    Applicant: CSL LIMITED

    Inventor： ANDREWS, Arna ,  PANOUSIS, Kosta ,  EMMRICH, Kerstin ,  WILSON, Michael ,  DOWER, Steve ,  HARDY, Mathew ,  HARTMAN, Dallas

    IPC: A61K38/36 ,  A61K38/37 ,  C07K14/745 ,  A61P7/04

    CPC classification number: C07K14/755 ,  A61K35/00 ,  A61K38/00 ,  C07K2319/31

    Abstract: The present invention provides a modified polypeptide which binds Factor VIII. The polypeptide comprises truncated von Willebrand Factor (VWF) which comprises a sequence as shown in SEQ ID NO:3 or a fragment thereof or a sequence 90% identical thereto, wherein the truncated VWF comprises at least one modification in comparison to SEQ ID NO: 3 in at least one position selected from the group consisting of SI, S3, LI 8, V42, S43, K149, N248, S279, V320, T325, Q395 and K418.

    Abstract translation: 本发明提供了结合因子VIII的修饰多肽。 所述多肽包含截短的血管性血友病因子（VWF），其包含如SEQ ID NO：3中所示的序列或其片段或与其90％相同的序列，其中所述截短的VWF包含与SEQ ID NO： 3在选自由S1，S3，L1 8，V42，S43，K149，N248，S279，V320，T325，Q395和K418组成的组中的至少一个位置中。

    FACTOR VIII WITH EXTENDED HALF-LIFE AND REDUCED LIGAND-BINDING PROPERTIES
    5.
    发明申请
    FACTOR VIII WITH EXTENDED HALF-LIFE AND REDUCED LIGAND-BINDING PROPERTIES 审中-公开
    因子VIII具有延长的半寿命和降低的配体结合特性

    公开(公告)号：WO2017096383A1

    公开(公告)日：2017-06-08

    申请号：PCT/US2016/064979

    申请日：2016-12-05

    Applicant: BAXALTA INCORPORATED ,  BAXALTA GMBH ,  TURECEK, Peter ,  SCHRENK, Gerald ,  SIEKMANN, Juergen

    Inventor： TURECEK, Peter ,  SCHRENK, Gerald ,  SIEKMANN, Juergen

    IPC: C07K14/755 ,  A61K47/61

    CPC classification number: C07K14/755 ,  A61K47/61

    Abstract: The invention relates to materials and methods of conjugating a water soluble polymer to an oxidized carbohydrate moiety of a therapeutic protein comprising contacting the oxidized carbohydrate moiety with an activated water soluble polymer under conditions that allow conjugation. More specifically, the present invention relates to a modified, recombinant Factor VIII (FVIII) with extended half-life and reduced ligand-binding properties.

    Abstract translation: 本发明涉及使水溶性聚合物与治疗性蛋白质的氧化碳水化合物部分缀合的材料和方法，包括在允许缀合的条件下使氧化的碳水化合物部分与活化的水溶性聚合物接触。 更具体地说，本发明涉及具有延长的半衰期和降低的配体结合性质的修饰的重组因子VIII（FVIII）。

    LIVER-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHODS OF USE THEREOF
    6.
    发明申请
    LIVER-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHODS OF USE THEREOF 审中-公开
    肝特异性构建体，因子VIII表达盒及其使用方法

    公开(公告)号：WO2017074526A1

    公开(公告)日：2017-05-04

    申请号：PCT/US2016/042099

    申请日：2016-07-13

    Applicant: SANGAMO BIOSCIENCES, INC.

    Inventor： RILEY, Brigit E.

    IPC: C12N15/00 ,  C12N15/113 ,  C12N15/63 ,  C12N15/85

    CPC classification number: A61K48/0058 ,  A61K38/00 ,  A61K38/37 ,  A61K45/06 ,  A61K48/00 ,  C07K14/755 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14171 ,  C12N2830/008

    Abstract: The present invention is in the field of gene therapy, particularly targeted delivery of transgene-encoding constructs to the liver for expression of beneficial (therapeutic) proteins. In particular, the invention relates to the treatment of hemophilias such as Hemophilia A. The present invention describes compositions and methods for expressing a transgene in a liver cell. The transgene may be expressed extrachromosomally (episomally) or may be integrated into the genome of the liver cell. In some embodiments, the transgene encodes a protein involved in the clotting cascade. In preferred embodiments, the transgene encodes a FVIII polypeptide.

    Abstract translation: 本发明属于基因治疗领域，特别是将转基因编码构建体靶向递送至肝脏以表达有益（治疗性）蛋白质。 具体而言，本发明涉及血友病例如血友病A的治疗。本发明描述了用于在肝细胞中表达转基因的组合物和方法。 转基因可以在染色体外表达（附加体）或可以整合到肝细胞的基因组中。 在一些实施方案中，转基因编码参与凝血级联的蛋白质。 在优选的实施方案中，转基因编码FVIII多肽。

    PRODUCTION OF OVERSIZED ADENO-ASSOCIATED VECTORS
    8.
    发明申请
    PRODUCTION OF OVERSIZED ADENO-ASSOCIATED VECTORS 审中-公开
    生产过度的腺苷相关载体

    公开(公告)号：WO2016164609A2

    公开(公告)日：2016-10-13

    申请号：PCT/US2016/026486

    申请日：2016-04-07

    Applicant: GENZYME CORPORATION

    Inventor： KYOSTIO-MOORE, Sirkka ,  SOUZA, David ,  VINCENT, Karen

    CPC classification number: C12N7/00 ,  C07K14/755 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14152

    Abstract: Provided herein are methods for producing an adeno-associated virus (AAV) particle containing an oversized recombinant AAV genome (e.g., greater than 4.7 kb). In some aspects, the invention provides AAV particles and AAV vectors comprising oversized rAAV genomes. Producer cell lines to produce AAV particles comprising oversized genomes are also provided.

    Abstract translation: 本文提供了用于产生含有超大重组AAV基因组（例如，大于4.7kb）的腺相关病毒（AAV）颗粒的方法。 在一些方面，本发明提供包含超大的rAAV基因组的AAV颗粒和AAV载体。 还提供了生产包含超大基因组的AAV颗粒的生产细胞系。

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