公开(公告)号：US12060349B2

公开(公告)日：2024-08-13

申请号：US17230385

申请日：2021-04-14

申请人： Genzyme Corporation

发明人： Elyse Bourque ,  Mario A. Cabrera-Salazar ,  Cassandra Celatka ,  Seng H. Cheng ,  Bradford Hirth ,  Andrew Good ,  Katherine Jancsics ,  John Marshall ,  Markus Metz ,  Ronald K. Scheule ,  Renato Skerlj ,  Yibin Xiang ,  Zhong Zhao ,  John Leonard ,  Thomas Natoli ,  Elina Makino ,  Herve Husson ,  Oxana Beskrovnaya

IPC分类号： C07D453/02 ,  A61K31/439 ,  A61K31/496 ,  A61K31/497 ,  A61K31/506 ,  A61K31/5377 ,  A61K31/55 ,  A61K31/551 ,  A61K45/06 ,  C07D471/08

CPC分类号： C07D453/02 ,  A61K31/439 ,  A61K31/496 ,  A61K31/497 ,  A61K31/506 ,  A61K31/5377 ,  A61K31/55 ,  A61K31/551 ,  A61K45/06 ,  C07D471/08

摘要： The invention relates to inhibitors of glucosylceramide synthase (GCS) of the Formula I: represented by the following structural formula




or a pharmaceutically acceptable salt thereof, wherein the variables are as defined herein, useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.

公开(公告)号：US11801311B2

公开(公告)日：2023-10-31

申请号：US16867261

申请日：2020-05-05

申请人： Genzyme Corporation

发明人： Carol A. Nelson ,  Bruce M. Wentworth ,  Ronald K. Scheule ,  Timothy E. Weeden ,  Nicholas P. Clayton

IPC分类号： C07H21/02 ,  C07H21/04 ,  A61K48/00 ,  C12N15/113

CPC分类号： A61K48/005 ,  C12N15/113 ,  C12N15/1137 ,  C12Y204/01011 ,  C12N2310/11 ,  C12N2310/3233 ,  C12N2310/3513 ,  C12N2320/33

摘要： The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase. AONs of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as Pompe disease. Also provided by the present disclosure are compositions comprising AONs, as well as methods of down regulating mRNA coding for glycogen synthase, methods for reducing glycogen synthase in skeletal and cardiac muscle, and methods for treating Pompe disease.

公开(公告)号：US20150190373A1

公开(公告)日：2015-07-09

申请号：US14464432

申请日：2014-08-20

申请人： Genzyme Corporation

发明人： Seng H. Cheng ,  Nelson S. Yew ,  Ronald K. Scheule ,  Hongmei Zhao

IPC分类号： A61K31/4025

CPC分类号： A61K31/4025 ,  A61K31/395 ,  A61K31/40 ,  Y10S514/866

摘要： The invention provides methods of treating a diabetic subject comprising administering a glucosylceramide synthase inhibitor to the subject.

摘要翻译： 本发明提供治疗糖尿病受试者的方法，包括向受试者施用葡萄糖神经酰胺合酶抑制剂。

公开(公告)号：US11008316B2

公开(公告)日：2021-05-18

申请号：US15490385

申请日：2017-04-18

申请人： Genzyme Corporation

发明人： Elyse Bourque ,  Mario A. Cabrera-Salazar ,  Cassandra Celatka ,  Seng H. Cheng ,  Bradford Hirth ,  Andrew Good ,  Katherine Jancsics ,  John Marshall ,  Markus Metz ,  Ronald K. Scheule ,  Renato Skerlj ,  Yibin Xiang ,  Zhong Zhao ,  John Leonard ,  Thomas Natoli ,  Elina Makino ,  Herve Husson ,  Oxana Beskrovnaya

IPC分类号： C07D453/02 ,  C07D471/08 ,  A61K31/439 ,  A61K31/496 ,  A61K31/497 ,  A61K31/506 ,  A61K31/5377 ,  A61K31/55 ,  A61K31/551 ,  A61K45/06

摘要： The invention relates to inhibitors of glucosylceramide synthase (GCS), such as Compound of Formula I, shown below, as defined herein, useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.

公开(公告)号：US09532976B2

公开(公告)日：2017-01-03

申请号：US14464432

申请日：2014-08-20

申请人： Genzyme Corporation

发明人： Seng H. Cheng ,  Nelson S. Yew ,  Ronald K. Scheule ,  Hongmei Zhao

IPC分类号： A61K31/40 ,  A61K31/4025 ,  A61K31/395

CPC分类号： A61K31/4025 ,  A61K31/395 ,  A61K31/40 ,  Y10S514/866

摘要： The invention provides methods of treating a diabetic subject comprising administering a glucosylceramide synthase inhibitor to the subject.

摘要翻译： 本发明提供治疗糖尿病受试者的方法，包括向受试者施用葡萄糖神经酰胺合酶抑制剂。

公开(公告)号：US10682423B2

公开(公告)日：2020-06-16

申请号：US15990463

申请日：2018-05-25

申请人： GENZYME CORPORATION

发明人： Carol A. Nelson ,  Bruce M. Wentworth ,  Ronald K. Scheule ,  Timothy E. Weeden ,  Nicholas P. Clayton

IPC分类号： A61K48/00 ,  C07H21/04 ,  C12N15/113

摘要： The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase. AONs of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as Pompe disease. Also provided by the present disclosure are compositions comprising AONs, as well as methods of down regulating mRNA coding for glycogen synthase, methods for reducing glycogen synthase in skeletal and cardiac muscle, and methods for treating Pompe disease.

公开(公告)号：US20150210681A1

公开(公告)日：2015-07-30

申请号：US14427275

申请日：2013-09-10

申请人： GENZYME CORPORATION

发明人： Elyse Bourque ,  Mario A. Cabrera-Salazar ,  Cassandra Celatka ,  Seng H. Cheng ,  Bradford Hirth ,  Andrew Good ,  Katherine Jancsics ,  John Marshall ,  Markus Metz ,  Ronald K. Scheule ,  Renato Skerlj ,  Yibin Xiang ,  Zhong Zhao ,  John Leonard ,  Thomas Natoli ,  Elina Makino ,  Herve Husson ,  Oxana Beskrovnaya

IPC分类号： C07D453/02 ,  C07D471/08 ,  A61K31/55 ,  A61K45/06 ,  A61K31/506 ,  A61K31/497 ,  A61K31/439 ,  A61K31/5377 ,  A61K31/496 ,  A61K31/551

CPC分类号： C07D453/02 ,  A61K31/439 ,  A61K31/496 ,  A61K31/497 ,  A61K31/506 ,  A61K31/5377 ,  A61K31/55 ,  A61K31/551 ,  A61K45/06 ,  C07D471/08

摘要： The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.

摘要翻译： 本发明涉及可用于单独或与酶替代疗法，囊性疾病和癌症治疗组合治疗代谢性疾病如溶酶体贮积病的葡萄糖神经酰胺合成酶（GCS）抑制剂。