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    MODIFIED FORMS OF PSEUDOMONAS EXOTOXIN A
    13.
    发明申请
    MODIFIED FORMS OF PSEUDOMONAS EXOTOXIN A 审中-公开

    公开(公告)号：US20160108377A1

    公开(公告)日：2016-04-21

    申请号：US14984006

    申请日：2015-12-30

    Applicant: Intrexon Corporation

    Inventor： Timothy David Jones ,  Francis Joseph Carr

    IPC: C12N9/10

    CPC classification number: C12N9/1077 ,  A61K38/00 ,  A61K39/00 ,  A61K39/02 ,  C07K14/21 ,  C07K16/2803 ,  C07K2317/622 ,  C12Y204/02036

    Abstract: Pseudomonas exotoxin A or “PE” is a 66 kD, highly potent, cytotoxic protein secreted by the bacterium Pseudomonas aeruginosa. Various forms of PE have been coupled to other proteins, such as antibodies, to generate therapeutically useful cytotoxin conjugates that selectively target cells of a desired phenotype (such as tumor cells). In the present invention, peptides spanning the sequence of an approximately 38 kD form of Pseudomonas exotoxin A protein were analyzed for the presence of immunogenic CD4+ T cell epitopes. Six immunogenic T cell epitopes were identified. Residues were identified within each epitope for introduction of targeted amino acid substitutions to reduce or prevent immunogenic T-cell responses in PE molecules which may be administered to a heterologous host.

    Methods and compositions for diagnosing disease
    14.
    发明授权
    Methods and compositions for diagnosing disease 有权
    用于诊断疾病的方法和组合物

    公开(公告)号：US09222121B2

    公开(公告)日：2015-12-29

    申请号：US14459480

    申请日：2014-08-14

    Applicant: Intrexon Corporation

    Inventor： Robert P. Beech ,  Thomas D. Reed ,  Robert Patzig

    IPC: A61K49/00 ,  C12Q1/00 ,  C12Q1/68 ,  C12Q1/66 ,  C12N15/63

    CPC classification number: C12Q1/6897 ,  C12N15/63 ,  C12N15/85 ,  C12Q1/66 ,  C12Q1/6883 ,  C12Q2600/158 ,  G01N2800/245

    Abstract: The present invention relates to methods and compositions for diagnosing a disease or disorder in a subject by introducing into cells of the subject a diagnostic gene switch construct and monitoring expression of a reporter gene. The invention further relates to methods and compositions for monitoring the progression of a disease or disorder or the effectiveness of a treatment for a disease or disorder.

    Abstract translation: 本发明涉及用于诊断受试者的疾病或病症的方法和组合物，该方法和组合物通过将诊断基因切换构建体导入受试者的细胞并监测报道基因的表达。 本发明还涉及用于监测疾病或病症进展或疾病或病症治疗有效性的方法和组合物。

    Crystalline Diacylhydrazine and the Use Thereof
    19.
    发明申请
    Crystalline Diacylhydrazine and the Use Thereof 有权
    结晶二酰肼及其用途

    公开(公告)号：US20150045441A1

    公开(公告)日：2015-02-12

    申请号：US14519408

    申请日：2014-10-21

    Applicant: Intrexon Corporation

    Inventor： Robert E. HORMANN ,  Inna Shulman ,  Eva Rödel ,  Rolf Hilfiker ,  Susan M. De Paul

    IPC: C07C243/38 ,  A61K31/166

    CPC classification number: C07C243/38 ,  A01N37/28 ,  A01N37/40 ,  A61K31/166 ,  A61K47/10 ,  A61K47/12 ,  A61K47/22 ,  A61K47/24 ,  C07B2200/07 ,  C07B2200/13 ,  C07C241/02 ,  C12N2710/10341 ,  C12N2830/002 ,  Y02A50/401 ,  Y02A50/409 ,  Y02A50/411 ,  Y02A50/423 ,  Y02A50/475 ,  Y10T428/2982

    Abstract: The present disclosure provides crystalline polymorphic and amorphous forms of (R)-3,5-dimethyl-benzoic acid N-(1-tert-butyl-butyl)-N′-(2-ethyl-3-methoxy-benzoyl)-hydrazide (Compound 1) or (S)-3,5-dimethyl-benzoic acid N-(1-tert-butyl-butyl)-N′-(2-ethyl-3-methoxy-benzoyl)-hydrazide (Compound 2). The present disclosure further provides compositions comprising crystalline polymorphic and amorphous forms of Compound 1 or Compound 2 and an excipient, methods of making crystalline polymorphic or amorphous forms of Compound 1 or Compound 2, and methods of using crystalline polymorphic or amorphous forms of Compound 1 or Compound 2 to regulate gene expression in a cell or in a subject.

    Abstract translation: 本公开提供（R）-3,5-二甲基 - 苯甲酸N-（1-叔丁基 - 丁基）-N' - （2-乙基-3-甲氧基 - 苯甲酰基） - 酰肼的结晶多晶型和无定形形式 （化合物1）或（S）-3,5-二甲基 - 苯甲酸N-（1-叔丁基 - 丁基）-N' - （2-乙基-3-甲氧基 - 苯甲酰基） - 酰肼（化合物2）。 本公开进一步提供了包含化合物1或化合物2的结晶多晶型和无定形形式的组合物和赋形剂，制备化合物1或化合物2的结晶多晶型或无定形形式的方法，以及使用化合物1的结晶多晶型或无定形形式的方法或 化合物2以调节细胞或受试者中的基因表达。

    Endoplasmic Reticulum Localization Signals
    20.
    发明申请
    Endoplasmic Reticulum Localization Signals 审中-公开
    内质网定位信号

    公开(公告)号：US20150018521A1

    公开(公告)日：2015-01-15

    申请号：US14194476

    申请日：2014-02-28

    Applicant: Intrexon Corporation

    Inventor： Thomas D. REED

    IPC: C07K14/47

    CPC classification number: C07K14/47 ,  A01K67/0275 ,  A01K2217/05 ,  A01K2227/105 ,  C07K2/00 ,  C07K2319/04 ,  C12N15/625 ,  C12N15/8509

    Abstract: The invention relates to cellular localization signals. In particular, the invention relates to endoplasmic reticulum localization signals in monomeric or multimeric form. The localization signals are utilized as research tools or are linked to therapeutics. Disclosed are methods of making and using polypeptides and modified polypeptides as signals to localize therapeutics, experimental compounds, peptides, proteins and/or other macromolecules to the endoplasmic reticulum of eukaryotic cells. The polypeptides of the invention optionally include linkage to reporters, epitopes and/or other experimental or therapeutic molecules. The invention also encompasses polynucleotides encoding the localization signals and vectors comprising these polynucleotides.

    Abstract translation: 本发明涉及细胞定位信号。 特别地，本发明涉及单体或多聚体形式的内质网定位信号。 本地化信号被用作研究工具或与治疗有关。 公开了制备和使用多肽和修饰的多肽作为将治疗剂，实验化合物，肽，蛋白质和/或其他大分子定位于真核细胞的内质网的信号的方法。 本发明的多肽任选地包括与记者，表位和/或其他实验或治疗分子的连接。 本发明还包括编码定位信号的多核苷酸和包含这些多核苷酸的载体。

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