公开(公告)号：US09315790B2

公开(公告)日：2016-04-19

申请号：US14026471

申请日：2013-09-13

申请人： TOKYO METROPOLITAN INSTITUTE OF MEDICAL SCIENCE ,  ALTIF LABORATORIES INC.

发明人： Hitoshi Sakuraba ,  Youichi Tajima ,  Ikuo Kawashima ,  Seiichi Aikawa ,  Fumiko Aikawa

IPC分类号： C12N9/40 ,  A61K38/47 ,  A61K48/00

CPC分类号： C12N9/2465 ,  A61K38/47 ,  A61K48/005

摘要： The present invention provides a pharmaceutical composition comprising a protein having α-galactosidase activity for treating Fabry disease, which causes no allergic side effect, which is highly stable in blood (plasma) and which can readily be taken up by a cell of an affected organ. The pharmaceutical composition for treating Fabry disease of the invention comprises, for example, a protein which acquires an α-galactosidase activity through alteration of the structure of the active site of wild-type human α-N-acetylgalactosaminidase.

摘要翻译： 本发明提供一种药物组合物，其包含具有α-半乳糖苷酶活性的蛋白质，用于治疗法布里病，其不引起过敏性副作用，其在血液（血浆）中高度稳定，并且可容易地被受影响器官的细胞摄取 。 用于治疗本发明的法布里病的药物组合物包括例如通过改变野生型人α-N-乙酰半乳糖胺酶的活性位点的结构而获得α-半乳糖苷酶活性的蛋白质。

公开(公告)号：US20140171493A1

公开(公告)日：2014-06-19

申请号：US14162135

申请日：2014-01-23

申请人： ALTIF LABORATORIES ,  Tokyo Metropolitan Institute of Medical Science

发明人： Hitoshi Sakuraba ,  Youichi Tajima ,  Mai Ito ,  Seiichi Aikawa ,  Fumiko Aikawa

IPC分类号： C12N9/40

CPC分类号： C12N9/2465 ,  A61K38/00 ,  C12N9/2402 ,  C12Y302/01022 ,  C12Y302/01049

摘要： The present invention provides, as an enzyme which can be used for enzyme replacement therapy for Fabry disease, a protein having α-galactosidase activity, which shows no allergic adverse side effect, shows a high stability in blood, and can be easily incorporated into a cell of an affected organ. The protein of the present invention is a protein which has acquired α-galactosidase activity by changing the structure of the active site of wild-type human α-N-acetylgalactosaminidase.

摘要翻译： 本发明提供作为法布里病的酶替代疗法的酶，不具有过敏性副作用的具有α-半乳糖苷酶活性的蛋白质在血液中显示高稳定性，并且可以容易地加入到 受影响器官的细胞。 本发明的蛋白质是通过改变野生型人α-N-乙酰半乳糖胺酶活性位点的结构而获得α-半乳糖苷酶活性的蛋白质。

公开(公告)号：US09193964B2

公开(公告)日：2015-11-24

申请号：US14162135

申请日：2014-01-23

申请人： TOKYO METROPOLITAN INSTITUTE OF MEDICAL SCIENCE ,  ALTIF LABORATORIES

发明人： Hitoshi Sakuraba ,  Youichi Tajima ,  Mai Ito ,  Seiichi Aikawa ,  Fumiko Aikawa

IPC分类号： A61K48/00 ,  A61K38/47 ,  A61K38/43 ,  C12N9/40 ,  C12P21/06 ,  C12P19/34 ,  C12N15/00 ,  C07H21/04 ,  C07K1/00 ,  C12N9/24 ,  A61K38/00

CPC分类号： C12N9/2465 ,  A61K38/00 ,  C12N9/2402 ,  C12Y302/01022 ,  C12Y302/01049

摘要： The present invention provides, as an enzyme which can be used for enzyme replacement therapy for Fabry disease, a protein having α-galactosidase activity, which shows no allergic adverse side effect, shows a high stability in blood, and can be easily incorporated into a cell of an affected organ. The protein of the present invention is a protein which has acquired α-galactosidase activity by changing the structure of the active site of wild-type human α-N-acetylgalactosaminidase.

摘要翻译： 本发明提供作为法布里病的酶替代疗法的酶，不具有过敏性副作用的具有α-半乳糖苷酶活性的蛋白质在血液中显示高稳定性，并且可以容易地加入到 受影响器官的细胞。 本发明的蛋白质是通过改变野生型人α-N-乙酰半乳糖胺酶活性位点的结构而获得α-半乳糖苷酶活性的蛋白质。