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1.发明授权
公开(公告)号：EP2245145B1
公开(公告)日：2016-12-14
申请号：EP09702074.7
申请日：2009-01-16
CPC分类号： C12N9/16 , A01K2217/075 , A01K2267/035 , A61K38/00 , C12Q1/34 , C12Q1/44 , C12Y301/06014 , G01N33/6893 , G01N2333/924 , G01N2400/40 , G01N2500/04 , G01N2800/04
摘要： This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the lysosomal sulfatase enzyme.
摘要翻译： 本发明提供活性高度磷酸化溶酶体硫酸酯酶的组合物，其药物组合物，生产和纯化这些溶酶体硫酸酯酶的组合物及其组合物及其在疾病和病症的诊断，预防或治疗中的用途，特别是溶酶体贮积病， 由溶酶体硫酸酯酶缺乏引起或与之相关。
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2.发明公开MANUFACTURE OF ACTIVE HIGHLY PHOSPHORYLATED HUMAN LYSOSOMAL SULFATASE ENZYMES AND USES THEREOF 有权HERSTELLUNG SUMMVER HOCHPHOSPHORYLIERTER MENSCHLICHER LYSOSOMEN-SULFATASEN UND VERWENDUNGEN DAVON
公开(公告)号：EP2245145A2
公开(公告)日：2010-11-03
申请号：EP09702074.7
申请日：2009-01-16
CPC分类号： C12N9/16 , A01K2217/075 , A01K2267/035 , A61K38/00 , C12Q1/34 , C12Q1/44 , C12Y301/06014 , G01N33/6893 , G01N2333/924 , G01N2400/40 , G01N2500/04 , G01N2800/04
摘要： This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the lysosomal sulfatase enzyme.
摘要翻译： 本发明提供活性高度磷酸化溶酶体硫酸酯酶的组合物，其药物组合物，生产和纯化这些溶酶体硫酸酯酶的组合物及其组合物及其在疾病和病症的诊断，预防或治疗中的用途，特别是溶酶体贮积病， 由溶酶体硫酸酯酶缺乏引起或与之相关。
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3.发明公开
公开(公告)号：EP3352787A1
公开(公告)日：2018-08-01
申请号：EP16778589.8
申请日：2016-09-23
IPC分类号： A61K39/12 , C07K14/755
CPC分类号： A61K38/37 , A61K39/12 , A61K47/02 , A61K47/10 , A61K47/26 , A61K48/0008 , A61K48/0075 , C07K14/755 , C12N7/00 , C12N2710/16044 , C12N2750/14121 , C12N2750/14141 , C12N2750/14143 , G01N33/6854 , G01N2333/015
摘要： The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.
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