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71. 发明申请

WO2014005019A2 CELLS FOR PRODUCING RECOMBINANT IDURONATE-2-SULFATASE 审中-公开
标题翻译：用于生产重组IDURONATE-2-SULFATASE的细胞
公开(公告)号：WO2014005019A2
公开(公告)日：2014-01-03
申请号：PCT/US2013/048571
申请日：2013-06-28
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： BOLGOG, Ferenc , HEARTLEIN, Mike
IPC分类号： C12N9/16
CPC分类号： C12N9/16 , C12Y301/06013
摘要： The present invention provides, among other things, methods and compositions for production of recombinant I2S protein with improved potency and activity using cells co- express I2S and FGE protein. In some embodiments, cells according to the present invention are engineered to simultaneously over-express recombinant I2S and FGE proteins. Cells according to the invention are adaptable to various cell culture conditions. In some embodiments, cells of the present invention adaptable to a large-scale suspension serum- free culture.
摘要翻译：本发明提供了用于使用共表达I2S和FGE蛋白的细胞生产具有改善的效力和活性的重组I2S蛋白的方法和组合物。在一些实施方案中，根据本发明的细胞被工程化以同时过度表达重组I2S和FGE蛋白。根据本发明的细胞适用于各种细胞培养条件。在一些实施方案中，本发明的细胞适用于大规模悬浮无血清培养物。

72. 发明申请

WO2013177264A1 ANTI-CCL2 ANTIBODIES FOR TREATMENT OF SCLERODERMA 审中-公开
标题翻译： ANTI-CCL2治疗SCLERODERMA的抗体
公开(公告)号：WO2013177264A1
公开(公告)日：2013-11-28
申请号：PCT/US2013/042196
申请日：2013-05-22
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： MARTINI, Paolo, G.V. , NATARAJAN, Madhusudan , HASLETT, Patrick, Anthony, John , SEYMOUR, Albert, Barnes
IPC分类号： A61K39/395 , C07K16/00 , C12P21/08
CPC分类号： C07K16/24 , A61K2039/505 , C07K2317/21 , C07K2317/24 , C07K2317/51 , C07K2317/515 , C07K2317/56 , C07K2317/92
摘要： The present invention provides, among other things, improved anti-CCL2 antibodies characterized with high affinity, potency, tissue selectivity and/or epitope specificity, and uses thereof, in particular, for treatment of scleroderma and related fibrotic and/or inflammatory diseases, disorders and conditions. In some embodiments, the present invention provides methods and compositions for treatment of scleroderma and related fibrotic and/or inflammatory diseases, disorders and conditions based on an anti-CCL2 antibody having an affinity of 10 -12 M or greater.
摘要翻译：本发明尤其提供了以高亲和力，效力，组织选择性和/或表位特异性为特征的改进的抗CCL2抗体及其用途，特别是用于治疗硬皮病和相关的纤维化和/或炎性疾病，疾病和条件。在一些实施方案中，本发明提供了用于治疗基于具有10-12M或更大亲和力的抗CCL2抗体的硬皮病和相关纤维化和/或炎性疾病，病症和病症的方法和组合物。

73. 发明申请

WO2013148323A1 METHODS OF ANALYZING AND PREPARING PROTEIN COMPOSITIONS 审中-公开
标题翻译：分析和制备蛋白质组合物的方法
公开(公告)号：WO2013148323A1
公开(公告)日：2013-10-03
申请号：PCT/US2013/032171
申请日：2013-03-15
申请人： SHIRE HUMAN GENETIC THERAPIES, INC. , LIN, Melanie , SALINAS, Paul , WU, Shiaw-lin
发明人： SHIRE HUMAN GENETIC THERAPIES, INC. , LIN, Melanie , SALINAS, Paul , WU, Shiaw-lin
IPC分类号： A61K38/46
CPC分类号： C12Q1/44 , G01N2560/00
摘要： Methods for analyzing protein samples of recombinant human arylsulfatase A(rhASA), having one or more disulfide linkages are described. In some instances, the samples are analyzed using enzyme digestion, e.g., multi-enzyme digestion and liquid chromatography-mass spectrometry (LC-MS). The proteins described herein can be glycosylated (e.g., fully or partially glycosylated) or non-glycosylated. The methods described herein can include one or more of enzymatic digestion, chromatography, and mass spectrometry, e.g., multi-enzyme digestion and/or liquid chromatography-mass spectrometry (LC-MS). In some embodiments, the mass spectrometry utilizes collision induced dissociation (CID) and/or electron transfer dissociation (ETD). For example, the mass spectrometry can utilize CID, ETD, and CID of the isolated charge-reduced ions (MS3), e.g., after ETD.
摘要翻译：描述了分析具有一个或多个二硫键的重组人芳基硫酸酯酶A（rhASA）的蛋白质样品的方法。在一些情况下，使用酶消化（例如多酶消化和液相色谱 - 质谱法（LC-MS））分析样品。本文所述的蛋白质可以被糖基化（例如完全或部分糖基化）或非糖基化。本文所述的方法可以包括酶消化，色谱法和质谱法中的一种或多种，例如多酶消化和/或液相色谱 - 质谱（LC-MS）。在一些实施方案中，质谱法利用碰撞诱导解离（CID）和/或电子转移离解（ETD）。例如，质谱可以利用分离的电荷还原离子（MS3）的CID，ETD和CID，例如在ETD之后。

74. 发明申请

WO2013148277A1 SUBCUTANEOUS ADMINISTRATION OF IDURONATE- 2-SULFATASE 审中-公开
标题翻译： IDURONATE-2-SULFATASE的不足管理
公开(公告)号：WO2013148277A1
公开(公告)日：2013-10-03
申请号：PCT/US2013/031662
申请日：2013-03-14
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： XIE, Hongsheng , FELICE, Brian , MCCAULEY, Thomas
IPC分类号： A61K38/00 , A61K38/43 , A61P25/00
CPC分类号： A61K38/465 , A61K9/0019 , A61K38/46 , C12Y301/06013
摘要： The present invention provides, among other things, compositions, kits and methods for subcutaneous delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention provides methods for treating Hunter syndrome by subcutaneous administration of a replacement iduronate-2-sulfatase (I2S) protein. In some embodiments, the present invention provides a kit comprising an arrangement of components for subcutaneously administering iduronate-2-sulfatase (12 S) protein.
摘要翻译：本发明尤其提供用于皮下递送溶酶体酶用于有效治疗溶酶体贮积病的组合物，试剂盒和方法。在一些实施方案中，本发明提供了通过皮下施用替代的阿魏酸-2-硫酸酯酶（I 2 S）蛋白来治疗亨特综合征的方法。在一些实施方案中，本发明提供了一种试剂盒，其包含用于皮下施用艾杜糖醛酸-2-硫酸酯酶（12S）蛋白质的组分排列。

75. 发明申请

WO2013138353A2 COMPOSITIONS AND METHODS FOR MODULATION OF ATXN3 EXPRESSION 审中-公开
标题翻译： ATXN3表达调控的组成和方法
公开(公告)号：WO2013138353A2
公开(公告)日：2013-09-19
申请号：PCT/US2013/030553
申请日：2013-03-12
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： UZCÁTEGUI, Nathalie , HEDTJÄRN, Maj , HANSEN, Jens, Bo, Rode
IPC分类号： A61K48/00
CPC分类号： C12N15/1137 , A61K9/0085 , C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/3519 , C12N2320/34
摘要： Disclosed are oligonucleotides which target and hybridize to nucleic acid molecules encoding ATXN3, leading to reduced expression of ATXN3, Reduction in the expression of aberrant ATXN3 is beneficial for the treatment of certain medical disorders, such as spinocerebellar ataxia 3.
摘要翻译：公开了与编码ATXN3的核酸分子靶向和杂交的寡核苷酸，导致ATXN3的表达降低，异常ATXN3的表达的降低有利于治疗某些医学病症，例如脊髓小脑性共济失调3。

76. 发明申请

WO2013130963A1 COMPOSITIONS AND METHODS FOR TREATING TYPE III GAUCHER DISEASE 审中-公开
标题翻译：用于治疗III型GAUCHER疾病的组合物和方法
公开(公告)号：WO2013130963A1
公开(公告)日：2013-09-06
申请号：PCT/US2013/028608
申请日：2013-03-01
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： CROMBEZ, Eric , BHIRANG, Kiran , COHN, Gabriel, Martin
IPC分类号： A61K38/46 , A61K38/47
CPC分类号： A61K38/47 , A61K9/0019 , C12Y302/01045 , G01N33/538 , G01N33/6854 , G01N2333/924 , G01N2800/044 , G01N2800/52
摘要： Methods and compositions for treating Gaucher disease are described.
摘要翻译：描述了治疗戈谢病的方法和组合物。

77. 发明申请

WO2013070760A1 BIOMARKERS FOR SANFILIPPO SYNDROME AND USES THEREOF 审中-公开
标题翻译： SANFILIPPO综合症的生物标志物及其用途
公开(公告)号：WO2013070760A1
公开(公告)日：2013-05-16
申请号：PCT/US2012/063935
申请日：2012-11-07
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： HASLETT, Patrick , RICHARD, Charlie
IPC分类号： C12M1/34
CPC分类号： G01N33/6893 , A61K38/46 , C12Q1/6883 , C12Q2600/118 , C12Q2600/158 , G01N2333/4753 , G01N2400/10 , G01N2440/14 , G01N2800/04 , G01N2800/2814 , G01N2800/2835 , G01N2800/52
摘要： The present invention provides biomarkers for efficient and accurate characterization of Sanfilippo syndrome. In particular, the present invention provides biomarkers differentially expressed in Sanfilippo syndrome. Those biomarkers, used alone or in combination, may permit more accurate robust characterization of Sanfilippo syndrome, resulting in more precise determination of the types and/or severity of the syndrome. In addition, inventive biomarkers according to the present invention can be used to effective monitor treatment response in Sanfilippo syndrome patients and/or to optimize treatment for Sanfilippo syndrome.
摘要翻译：本发明提供用于有效和准确地表征Sanfilippo综合征的生物标志物。特别地，本发明提供了在Sanfilippo综合征中差异表达的生物标志物。单独使用或组合使用的那些生物标志物可以使Sanfilippo综合征更准确地稳定地表征，从而更准确地确定综合征的类型和/或严重程度。此外，根据本发明的本发明的生物标志物可用于有效监测Sanfilippo综合征患者的治疗反应和/或优化Sanfilippo综合征的治疗。

78. 发明申请

WO2011163651A2 METHODS AND COMPOSITIONS FOR CNS DELIVERY OF β-GALACTOCEREBROSIDASE 审中-公开
标题翻译： CNS递送β-GALACTOCEREBROSIDASE的方法和组合物
公开(公告)号：WO2011163651A2
公开(公告)日：2011-12-29
申请号：PCT/US2011/041927
申请日：2011-06-25
申请人： SHIRE HUMAN GENETIC THERAPIES, INC. , SALAMAT-MILLER, Nazila , TAYLOR, Katherine , MANNING, Ken , ZHU, Gaozhong
发明人： SALAMAT-MILLER, Nazila , TAYLOR, Katherine , MANNING, Ken , ZHU, Gaozhong
IPC分类号： A61K38/47 , A61P25/00 , A61P25/28
CPC分类号： A61K9/0019 , A61K9/0085 , A61K9/19 , A61K38/465 , A61K38/47 , A61K47/02 , A61K47/26 , C07K14/65 , C12N9/2402 , C12N9/2437 , C12Y301/06013 , C12Y302/01046
摘要： The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an B-Galactocerebrosidase protein, salt, and a polysorbate surfactant for the treatment of GLD Disease.
摘要翻译：本发明尤其提供了用于CNS递送溶酶体酶以有效治疗溶酶体贮积病的组合物和方法。在一些实施方案中，本发明包括用于直接CNS鞘内施用的稳定制剂，其包含用于治疗GLD疾病的B-半乳糖脑苷脂酶蛋白质，盐和聚山梨醇酯表面活性剂。

79. 发明申请

WO2022197789A1 POLYCYCLIC INHIBITORS OF PLASMA KALLIKREIN 审中-公开
公开(公告)号：WO2022197789A1
公开(公告)日：2022-09-22
申请号：PCT/US2022/020527
申请日：2022-03-16
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： PAPAIOANNOU, Nikolaos , TRAVINS, Jeremy Mark , FINK, Sarah Jocelyn , ELLARD, John Mark , RAE, Alastair , RANKIN, Stuart Shane , CHAPMAN, Robert Stuart Laurie
IPC分类号： C07D471/04 , C07D487/04 , A61P9/00 , A61P29/00 , A61K31/437
摘要： The present invention provides compounds and compositions thereof which are useful as inhibitors of plasma kallikrein and which exhibit desirable characteristics for the same.

80. 发明申请

WO2022197763A1 INHIBITORS OF PLASMA KALLIKREIN 审中-公开
公开(公告)号：WO2022197763A1
公开(公告)日：2022-09-22
申请号：PCT/US2022/020491
申请日：2022-03-16
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： PAPAIOANNOU, Nikolaos , TRAVINS, Jeremy Mark , FINK, Sarah Jocelyn , ELLARD, John Mark , RAE, Alastair
IPC分类号： A61P9/00 , A61P29/00 , C07D417/12 , C07D417/14 , C07D471/04 , C07D401/14 , C07D403/06 , C07D403/12 , C07D403/14 , C07D487/04 , C07D495/04 , A61K31/437
摘要： The present invention provides compounds and compositions thereof which are useful as inhibitors of plasma kallikrein and which exhibit desirable characteristics for the same.

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