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公开(公告)号：EP3558947A2
公开(公告)日：2019-10-30
申请号：EP17829884.0
申请日：2017-12-22
发明人： LUEDTKE, Gregory , BHAGWAT, Shripad
IPC分类号： C07D223/04 , C07D241/04 , C07D243/08 , C07D487/10 , C07D205/04 , C07D498/10 , C07D211/12 , C07D211/14 , A61K31/553 , A61K31/4965 , A61K31/44 , A61K31/55 , A61K31/397 , A61K31/4427 , A61P25/00
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113.发明公开
公开(公告)号：EP3419651A1
公开(公告)日：2019-01-02
申请号：EP17711041.8
申请日：2017-02-24
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114.发明授权N-(1-HYDROXY-3-(PYRROLIDINYL)PROPAN-2-YL)PYRROLIDINE-3-CARBOXAMIDE DERIVATIVES AS GLUCOSYLCERAMIDE SYNTHASE INHIBITORS 有权作为葡萄糖酰胺合成酶抑制剂的N-（1-羟基-3-（吡咯烷基）丙-2-基）吡咯烷-3-羧酰胺衍生物
公开(公告)号：EP3063141B1
公开(公告)日：2018-02-28
申请号：EP14802535.6
申请日：2014-10-28
发明人： WANG, Bing
IPC分类号： C07D403/12 , C07D401/14 , C07D405/14 , C07D409/14 , C07D417/14 , A61K31/4025 , A61P3/00 , A61P35/00 , A61P39/00
CPC分类号： C07D207/16 , C07D319/16 , C07D401/04 , C07D401/12 , C07D401/14 , C07D403/12 , C07D405/12 , C07D405/14 , C07D409/04 , C07D409/12 , C07D409/14 , C07D417/14 , C07D471/04
摘要： Described herein are compounds of Formula I, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and compounds I for use to treat or prevent diseases or conditions associated with the enzyme glucosylceramide synthase (GCS).
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公开(公告)号：EP3272773A1
公开(公告)日：2018-01-24
申请号：EP17182845.2
申请日：2009-05-07
发明人： LEBOWITZ, Jonathan , MAGA, John
IPC分类号： C07K19/00 , C07K14/475
摘要： The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology. Among other things, the present invention provides methods and compositions for targeting lysosomal enzymes to lysosomes using furin-resistant lysosomal targeting peptides. The present invention also provides methods and compositions for targeting lysosomal enzymes to lysosomes using a lysosomal targeting peptide that has reduced or diminished binding affinity for the insulin receptor.
摘要翻译： 基于GILT技术，本发明提供了用于有效溶酶体靶向的进一步改进的组合物和方法。 除此之外，本发明提供了使用弗林蛋白酶抗性溶酶体靶向肽将溶酶体酶靶向溶酶体的方法和组合物。 本发明还提供了使用对胰岛素受体具有降低或降低的结合亲和力的溶酶体靶向肽将溶酶体酶靶向溶酶体的方法和组合物。
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116.发明公开
公开(公告)号：EP3232193A3
公开(公告)日：2017-12-13
申请号：EP17162060.2
申请日：2009-12-31
CPC分类号： C12Q1/527 , A61K31/702 , C07H3/04 , C07H3/06 , C07H5/06 , C07H7/033 , C07H11/00 , C07H13/04 , G01N33/66 , G01N2333/988 , G01N2400/40 , G01N2800/042 , G01N2800/52 , G01N2800/7071
摘要： Provided herein are processes for detecting oligosaccharides in a biological sample. In specific instances, the biological sample is provided from an individual suffering from a disorder associated with abnormal glycosaminoglycan accumulation.
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117.发明公开
公开(公告)号：EP3204009A1
公开(公告)日：2017-08-16
申请号：EP15848965.8
申请日：2015-10-08
IPC分类号： A61K31/506 , A61K31/5377 , C07D413/14
CPC分类号： A61K31/506 , A61K45/06 , A61P25/28 , C07D401/14 , C07D403/04 , C07D403/14 , C07D405/14 , C07D413/14 , C07D471/04 , A61K2300/00
摘要： Described herein are compounds of Formula I, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or conditions in need of inhibition of heparan sulfate biosynthesis.
摘要翻译： 本文描述了式I的化合物，制备此类化合物的方法，含有此类化合物的药物组合物和药物，以及使用此类化合物治疗或预防需要抑制硫酸乙酰肝素生物合成的疾病或病症的方法。
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公开(公告)号：EP2279210B1
公开(公告)日：2017-04-12
申请号：EP09743707.3
申请日：2009-05-07
发明人： LEBOWITZ, Jonathan, H. , MAGA, John
IPC分类号： C07K19/00 , C07K14/475
CPC分类号： A61K38/47 , C07K14/65 , C07K2319/06 , C07K2319/33 , C12N9/2408 , C12Y302/0102
摘要： The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology. Among other things, the present invention provides methods and compositions for targeting lysosomal enzymes to lysosomes using furin-resistant lysosomal targeting peptides. The present invention also provides methods and compositions for targeting lysosomal enzymes to lysosomes using a lysosomal targeting peptide that has reduced or diminished binding affinity for the insulin receptor.
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119.发明授权MANUFACTURE OF ACTIVE HIGHLY PHOSPHORYLATED HUMAN N-ACETYLGALACTOSAMINE-6-SULFATASE AND USES THEREOF 有权生产和资产使用，HOCHPHOSPHORYLIERTEN人N-乙酰-6-硫酸酯酶
公开(公告)号：EP2595650B1
公开(公告)日：2016-11-16
申请号：EP11751689.8
申请日：2011-07-22
CPC分类号： C12N9/16 , A61K9/08 , A61K38/00 , A61K38/465 , C12Y301/06004
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公开(公告)号：EP3075386A1
公开(公告)日：2016-10-05
申请号：EP16153694.1
申请日：2010-06-17
发明人： LEBOWITZ, Jonathan , CHANG, Byeong
摘要： The present invention provides improved formulations for lysosomal enzymes useful for enzyme replacement therapy. Among other things, the present invention provides formulations that preserve or enhance the stability and/or efficacy of a lysosomal enzyme such as acid alpha-glucosidase.
摘要翻译： 本发明提供了用于酶替代疗法的溶酶体酶的改进制剂。 其中，本发明提供了保持或增强溶酶体酶如酸性α-葡糖苷酶的稳定性和/或功效的制剂。
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