公开(公告)号：US20150210681A1

公开(公告)日：2015-07-30

申请号：US14427275

申请日：2013-09-10

Applicant: GENZYME CORPORATION

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

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

Abstract: 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.

Abstract translation: 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。

公开(公告)号：US11008316B2

公开(公告)日：2021-05-18

申请号：US15490385

申请日：2017-04-18

Applicant: Genzyme Corporation

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

Abstract: 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：US12060349B2

公开(公告)日：2024-08-13

申请号：US17230385

申请日：2021-04-14

Applicant: Genzyme Corporation

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

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

Abstract: 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.