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公开(公告)号：US08492338B2
公开(公告)日：2013-07-23
申请号：US13481244
申请日：2012-05-25
CPC分类号： A61K38/30 , A61K38/47 , A61K47/551 , A61K47/6425 , C07K14/65 , C07K2319/00 , C07K2319/02 , C07K2319/50 , C07K2319/61 , C07K2319/75 , C12N15/62 , G01N2333/924 , A61K2300/00
摘要： Targeted therapeutics that localize to a specific subcellular compartment such as the lysosome are provided. The targeted therapeutics include a therapeutic agent and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.
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公开(公告)号：US07629309B2
公开(公告)日：2009-12-08
申请号：US10981267
申请日：2004-11-03
CPC分类号： A61K38/30 , A61K38/47 , A61K47/551 , A61K47/6425 , C07K14/65 , C07K2319/00 , C07K2319/02 , C07K2319/50 , C07K2319/61 , C07K2319/75 , C12N15/62 , G01N2333/924 , A61K2300/00
摘要： Targeted therapeutics that localize to a specific subcellular compartment such as the lysosome are provided. The targeted therapeutics include a therapeutic agent and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.
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公开(公告)号：US20120295327A1
公开(公告)日：2012-11-22
申请号：US13481244
申请日：2012-05-25
CPC分类号： A61K38/30 , A61K38/47 , A61K47/551 , A61K47/6425 , C07K14/65 , C07K2319/00 , C07K2319/02 , C07K2319/50 , C07K2319/61 , C07K2319/75 , C12N15/62 , G01N2333/924 , A61K2300/00
摘要： Targeted therapeutics that localize to a specific subcellular compartment such as the lysosome are provided. The targeted therapeutics include a therapeutic agent and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.
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公开(公告)号：US08207114B2
公开(公告)日：2012-06-26
申请号：US12610596
申请日：2009-11-02
CPC分类号： A61K38/30 , A61K38/47 , A61K47/551 , A61K47/6425 , C07K14/65 , C07K2319/00 , C07K2319/02 , C07K2319/50 , C07K2319/61 , C07K2319/75 , C12N15/62 , G01N2333/924 , A61K2300/00
摘要： Targeted therapeutics that localize to a specific subcellular compartment such as the lysosome are provided. The targeted therapeutics include a therapeutic agent and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.
摘要翻译： 提供了定位于特定亚细胞区室（例如溶酶体）的靶向治疗剂。 靶向治疗剂包括治疗剂和靶向部分，其结合细胞外表面上的受体，允许靶向治疗剂在受体内化时适当的亚细胞定位。 还提供了与本发明的实践有关的核酸，细胞和方法。
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公开(公告)号：US20100075906A1
公开(公告)日：2010-03-25
申请号：US12610596
申请日：2009-11-02
CPC分类号： A61K38/30 , A61K38/47 , A61K47/551 , A61K47/6425 , C07K14/65 , C07K2319/00 , C07K2319/02 , C07K2319/50 , C07K2319/61 , C07K2319/75 , C12N15/62 , G01N2333/924 , A61K2300/00
摘要： Targeted therapeutics that localize to a specific subcellular compartment such as the lysosome are provided. The targeted therapeutics include a therapeutic agent and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.
摘要翻译： 提供了定位于特定亚细胞区室（例如溶酶体）的靶向治疗剂。 靶向治疗剂包括治疗剂和靶向部分，其结合细胞外表面上的受体，允许靶向治疗剂在受体内化时适当的亚细胞定位。 还提供了与本发明的实践有关的核酸，细胞和方法。
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公开(公告)号：US08784833B2
公开(公告)日：2014-07-22
申请号：US13007772
申请日：2011-01-17
申请人： William S. Sly , Jeffrey H. Grubb , Shunjl Tomatsu , Adriana Maria Monta{umlaut over (n)}o Suarez , Hirotaka Oikawa
发明人： William S. Sly , Jeffrey H. Grubb , Shunjl Tomatsu , Adriana Maria Monta{umlaut over (n)}o Suarez , Hirotaka Oikawa
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： Transplacental enzyme replacement therapy (ERT) is disclosed for deficiency of a polypeptide such as a tissue-nonspecific alkaline phosphatase (TNSALP) by administering a pharmaceutical composition that comprises a water-soluble TNSALP portion, e.g., C-terminus-truncated TNSALP peptide-bonded to an IgG1 antibody Fc portion, to a pregnant animal whose fetus or embryo is in need of such therapy. Also contemplated is a method for treating a metabolic disorder, such as HPP, in a fetus or embryo where a fusion protein that comprises a Fc fragment of an IgG1 antibody peptide-bonded to TNSALP is administered to a pregnant mother. 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： 公开了通过施用包含水溶性TNSALP部分的药物组合物，例如C末端截短的TNSALP肽键合的多肽如TNSALP的组合非特异性碱性磷酸酶（TNSALP）的缺陷的胎盘期替代疗法（ERT） 到一种IgG1抗体Fc部分，其胎儿或胚胎需要这种治疗的怀孕动物。 还考虑了在胎儿或胚胎中治疗诸如HPP的代谢紊乱的方法，其中将包含与TNSALP结合的IgG1抗体的Fc片段的融合蛋白质施用于孕妇。 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。
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公开(公告)号：US20130011381A1
公开(公告)日：2013-01-10
申请号：US13455108
申请日：2012-04-24
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。
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公开(公告)号：US20090041741A1
公开(公告)日：2009-02-12
申请号：US12042601
申请日：2008-03-05
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。
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9.发明授权公开(公告)号：US08226940B2
公开(公告)日：2012-07-24
申请号：US12714534
申请日：2010-02-28
申请人： Shunji Tomatsu , Adriana Montano , Tatsuo Nishioka , Jeffrey H. Grubb , William S. Sly , Monica A. Gutierrez , Amelia Ortigoza Rodriguez, legal representative
发明人： Shunji Tomatsu , Adriana Montano , Tatsuo Nishioka , Jeffrey H. Grubb , William S. Sly , Monica A. Gutierrez
CPC分类号： C12N15/625 , A61K38/00 , C07K2319/02 , C07K2319/31 , C12N9/2402 , C12N9/96 , C12Y302/01031
摘要： The present invention provides a polypeptide therapeutic agent, useful in enzyme replacement therapy, with increased therapeutic benefits for the central nervous system. The invention provides a method of enhancing the effect of a polypeptide or protein on the central nervous system by the attachment of a short acidic amino acid sequence. Specifically the inventors disclose the attachment of a 4-15 acidic amino acid sequence to human β-glucuronidase by construction of a fusion protein. This molecule is useful in the treatment of type VII mucopolysaccharidosis when administered to a patient.
摘要翻译： 本发明提供了可用于酶替代疗法的多中心治疗剂，对中枢神经系统具有增加的治疗益处。 本发明提供了通过连接短酸性氨基酸序列增强多肽或蛋白质对中枢神经系统的作用的方法。 具体地，本发明人通过构建融合蛋白来公开了将4-15个酸性氨基酸序列与人类 - 葡萄糖醛酸苷酶的连接。 当给予患者时，该分子可用于治疗VII型粘多糖贮积病。
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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。
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