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

申请人： BioMarin Pharmaceutical Inc.

发明人： KOPPAKA, Vish ,  VELLARD, Michel, Claude ,  OKHAMAFE, Augustus, O. ,  ARAYA, Kidisti

IPC分类号： A61K38/46 ,  C12N9/16

CPC分类号： C12N9/16 ,  A61K9/08 ,  A61K38/00 ,  A61K38/465 ,  C12Y301/06004

公开(公告)号：EP0750511B1

公开(公告)日：2003-06-11

申请号：EP95914060.9

申请日：1995-03-15

申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA

发明人： ROSEN, Steven D. ,  HEMMERICH, Stefan ,  IMAI, Yasuyuki, Univ. of Tokyo

IPC分类号： A61K38/46 ,  A61K38/47 ,  A61K33/00 ,  A61K33/04 ,  A61P29/00

CPC分类号： C07K14/70596 ,  A61K33/04 ,  A61K33/20 ,  A61K38/465 ,  C12Y301/06004 ,  C12Y301/06014 ,  A61K2300/00

公开(公告)号：EP0750511A4

公开(公告)日：1999-05-06

申请号：EP95914060

申请日：1995-03-15

申请人： UNIV CALIFORNIA

发明人： ROSEN STEVEN D ,  HEMMERICH STEFAN ,  IMAI YASUYUKI

IPC分类号： C12N9/14 ,  A61K33/04 ,  A61K38/45 ,  A61K38/46 ,  A61P1/00 ,  A61P3/08 ,  A61P11/00 ,  A61P17/00 ,  A61P29/00 ,  A61P37/00 ,  C07K14/705 ,  C12N9/24 ,  A61K38/47

CPC分类号： C07K14/70596 ,  A61K33/04 ,  A61K33/20 ,  A61K38/465 ,  C12Y301/06004 ,  C12Y301/06014 ,  A61K2300/00

公开(公告)号：EP2595650A2

公开(公告)日：2013-05-29

申请号：EP11751689.8

申请日：2011-07-22

申请人： BioMarin Pharmaceutical Inc.

发明人： KOPPAKA, Vish ,  VELLARD, Michel, Claude ,  OKHAMAFE, Augustus, O. ,  ARAYA, Kidisti

IPC分类号： A61K38/46 ,  C12N9/16

CPC分类号： C12N9/16 ,  A61K9/08 ,  A61K38/00 ,  A61K38/465 ,  C12Y301/06004

摘要： This invention provides compositions of active highly phosphorylated human N- acetylgalactosamine-6-sulfatase (GALNS), and pharmaceutical compositions and formulations thereof, methods of producing and purifying GALNS, and its 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 GALNS enzyme,
e.g. , Mucopolysaccharidosis IVa (MPS IVa or Morquio A syndrome).

摘要翻译： 本发明提供活性高度磷酸化的人N-乙酰半乳糖胺-6-硫酸酯酶（GALNS）的组合物及其药物组合物及其制剂，制备和纯化GALNS的方法及其在疾病和病症的诊断，预防或治疗中的用途， 包括特别是由GALNS酶的缺陷引起或与之相关的溶酶体贮积病，例如粘多糖贮积症IVa（MPS IVa或Morquio A综合征）。

公开(公告)号：EP0750511A1

公开(公告)日：1997-01-02

申请号：EP95914060.0

申请日：1995-03-15

申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA

发明人： ROSEN, Steven D. ,  HEMMERICH, Stefan ,  IMAI, Yasuyuki, Univ. of Tokyo

IPC分类号： C12N9 ,  A61K33 ,  A61K38 ,  A61P1 ,  A61P3 ,  A61P11 ,  A61P17 ,  A61P29 ,  A61P37 ,  C07K14

CPC分类号： C07K14/70596 ,  A61K33/04 ,  A61K33/20 ,  A61K38/465 ,  C12Y301/06004 ,  C12Y301/06014 ,  A61K2300/00

摘要： Compositions are administered to a patient (preferably by injection and locally) to treat a variety of conditions including inflammation associated with trauma and with certain aspects of diseases such as rheumatoid arthritis, psoriasis, insulin-dependent diabetes, cutaneous lymphomas, duodenal ulcer, chronic proctitis, lymphocytic thyroiditis, hemorrhagic shock, reperfusion injury during transplantation and multiple sclerosis. The compositions are pharmaceutically acceptable injectable formulations which include an active component in a pharmaceutically acceptable carrier. The active component is a chlorate or selenate which inhibits the natural biochemical sulfation process and/or a sulfatase enzyme which removes a sulfate from a specific position of a saccharide molecule which makes up a part of a natural ligand for L-selectin. Removal of the sulfate from the ligand hinders the ability of the ligand to bind to its natural receptor (i.e. L-selectin) and thereby hinders a biochemical chain of events which results in inflammation.