利索-全球专利检索

  1. 登录
  2. 注册

搜索结果

12 条记录 (耗时 0.034 秒)

    PRENATAL ENZYME REPLACEMENT THERAPY
    3.
    发明申请
    PRENATAL ENZYME REPLACEMENT THERAPY 有权
    PRENATAL ENZYME替代治疗

    公开(公告)号：US20110300143A1

    公开(公告)日：2011-12-08

    申请号：US13007772

    申请日：2011-01-17

    申请人： William S. Sly ,  Jeffrey H. Grubb ,  Shunji Tomatsu ,  Adriana Maria Montaño Suarez ,  Hirotaka Oikawa

    发明人： William S. Sly ,  Jeffrey H. Grubb ,  Shunji Tomatsu ,  Adriana Maria Montaño Suarez ,  Hirotaka Oikawa

    IPC分类号： A61K39/395 ,  C12N9/16 ,  A61P3/00 ,  C07H21/04

    CPC分类号： C07K19/00 ,  A61K38/00 ,  A61K38/43 ,  C07K2319/00 ,  C07K2319/30 ,  C12N9/14 ,  C12N9/2402 ,  C12Y302/01031 ,  Y10S424/809 ,  Y10S530/866

    摘要： The invention contemplates transplacental enzyme replacement therapy (ERT) for deficiency of a polypeptide such as a tissue-nonspecific alkaline phosphatase (TNSALP) by administering a before-described pharmaceutical composition to a pregnant animal whose fetus or embryo is in need of such therapy. The fusion protein of such a composition comprises a water-soluble TNSALP portion, e.g., C-terminus-truncated TNSALP peptide-bonded to an IgG1 antibody Fc portion.The invention also contemplates a method for treating a metabolic disorder, such as HPP, in a fetus or embryo were a protein is administered to a pregnant mother. The fusion protein comprises a Fc fragment of an IgG1 antibody peptide-bonded to TNSALP. The protein crosses the placenta of the mother and enters the fetal blood stream. The protein is taken up into fetal tissue such that the TNSALP restores normal metabolic activity in the fetus.

    摘要翻译： 本发明通过将前述药物组合物给予胎儿或胚胎需要这种治疗的怀孕动物，考虑用于缺乏多肽如组织非特异性碱性磷酸酶（TNSALP）的跨胎盘酶替代疗法（ERT）。 这种组合物的融合蛋白包含水溶性TNSALP部分，例如与IgG1抗体Fc部分结合的C末端截短的TNSALP肽。 本发明还考虑了在胎儿或胚胎中治疗代谢性疾病如HPP的方法，即将蛋白质施用于怀孕母亲。 融合蛋白包含与TNSALP结合的IgG1抗体肽的Fc片段。 蛋白质穿过母体的胎盘并进入胎儿血液流。 蛋白质被吸收到胎儿组织中，使得TNSALP恢复胎儿的正常代谢活性。

    MODIFIED ENZYME TREATMENT METHOD
    5.
    发明申请
    MODIFIED ENZYME TREATMENT METHOD 审中-公开
    改良酶处理方法

    公开(公告)号：US20130011381A1

    公开(公告)日：2013-01-10

    申请号：US13455108

    申请日：2012-04-24

    申请人： William S. Sly ,  Jeffrey H. Grubb ,  Carole A. Vogler

    发明人： William S. Sly ,  Jeffrey H. Grubb ,  Carole A. Vogler

    IPC分类号： A61K38/47 ,  A61K38/43

    CPC分类号： C12Y302/01031 ,  C12N9/2402

    摘要： Disclosed is a method for the treatment of lysosomal storage disease in mammals wherein the mammal is administered a therapeutically effective amount of an isolated, modified recombinant β-glucuronidase whereby said storage diseased disease is relieved in the brain and visceral organs of the mammal. There is also disclosed an isolated, modified recombinant β-glucuronidase wherein the modification is having its carbohydrate moieties chemically modified so as to reduce its activity with respect to mannose and mannose 6-phosphate cellular delivery system while retaining enzymatic activity. Also disclosed are other lysosomal enzymes within the scope of the invention.

    摘要翻译： 公开了一种用于治疗哺乳动物溶酶体贮积病的方法，其中哺乳动物被施用治疗有效量的分离的，修饰的重组葡萄糖醛酸糖苷酶，由此在哺乳动物的脑和内脏器官中缓解所述贮存疾病。 还公开了一种分离的，修饰的重组葡萄糖醛酸糖苷酶，其中修饰具有化学修饰的其碳水化合物部分，以便在保留酶活性的同时降低其对甘露糖和甘露糖6-磷酸细胞递送系统的活性。 还公开了本发明范围内的其它溶酶体酶。

    MODIFIED ENZYME AND TREATMENT METHOD
    6.
    发明申请
    MODIFIED ENZYME AND TREATMENT METHOD 审中-公开
    改良的酶和处理方法

    公开(公告)号：US20090041741A1

    公开(公告)日：2009-02-12

    申请号：US12042601

    申请日：2008-03-05

    申请人： William S. Sly ,  Jeffrey H. Grubb ,  Carole A. Vogler

    发明人： William S. Sly ,  Jeffrey H. Grubb ,  Carole A. Vogler

    IPC分类号： A61K38/45 ,  C12N9/24 ,  A61K38/47 ,  C12N9/00 ,  A61P43/00 ,  C12N9/16 ,  C12N9/10

    CPC分类号： C12Y302/01031 ,  C12N9/2402

    摘要： There is disclosed an isolated, modified recombinant β-glucuronidase wherein the modification is having its carbohydrate moeties chemically modified so as to reduce its activity with respect to mannose and mannose 6-phosphate cellular delivery system while retaining enzymatic activity Also disclosed are methods for the treatment of lysosomal storage disease in mammals wherein the mammal is administered a therapeutically effective amount of isolated, modified recombinant β-glucuronidase whereby said storage diseased is relieved in the brain and visceral organs of the mammal. Also disclosed are other lysosomal enzymes within the scope of the invention.

    摘要翻译： 公开了一种分离的，修饰的重组β-葡糖醛酸糖苷酶，其中所述修饰具有化学修饰的其碳水化合物，以便在保留酶活性的同时降低其对甘露糖和甘露糖-6-磷酸细胞递送系统的活性。还公开了治疗方法 的哺乳动物中的溶酶体贮积病，其中哺乳动物被施用治疗有效量的分离的，修饰的重组β-葡糖醛酸糖苷酶，由此所述贮存患者在哺乳动物的脑和内脏器官中释放。 还公开了本发明范围内的其它溶酶体酶。

    Chimeral polypeptide composition for cross-placenta delivery
    7.
    发明授权
    Chimeral polypeptide composition for cross-placenta delivery 有权
    用于跨胎盘递送的多肽组合物

    公开(公告)号：US07871624B2

    公开(公告)日：2011-01-18

    申请号：US11769693

    申请日：2007-06-27

    申请人： Jeffrey H. Grubb ,  William S. Sly

    发明人： Jeffrey H. Grubb ,  William S. Sly

    IPC分类号： A61K39/00

    CPC分类号： C12Y302/01031 ,  A61K38/00 ,  C07K2319/30 ,  C12N9/2402

    摘要： The invention is directed to chimeral fusion proteins having an IgG1 antibody Fc portion and a lysosomal storage enzyme, particularly a Fc-GUS fusion protein useful in treating Sly's disease in an embryo or fetus. The invention is also directed to methods of treating in born errors of metabolism, particularly Sly's disease, in a fetus by delivering to a pregnant mother a Fc-MPS emzyme fusion protein.

    摘要翻译： 本发明涉及具有IgG1抗体Fc部分和溶酶体储存酶，特别是可用于治疗胚胎或胎儿中的Sly氏病的Fc-GUS融合蛋白的嵌合融合蛋白。 本发明还涉及通过向孕妇输送Fc-MPS酶融合蛋白来在胎儿中治疗出生的代谢错误，特别是Sly's疾病的方法。