公开(公告)号：US20140371460A1

公开(公告)日：2014-12-18

申请号：US14471958

申请日：2014-08-28

Applicant: Genzyme Corporation

Inventor： Elyse BOURQUE ,  Cassandra CELATKA ,  Bradford HIRTH ,  Markus METZ ,  Zhong ZHAO ,  Renato SKLERJ ,  Yibin XIANG ,  Katherine JANCSICS ,  John MARSHALL ,  Seng CHENG ,  Ronald SCHEULE ,  Mario CABRERA-SALAZAR ,  Andrew GOOD

IPC: C07D453/02

CPC classification number: C07D487/08 ,  A61K31/439 ,  A61K31/454 ,  A61K31/46 ,  A61K31/506 ,  A61K31/551 ,  A61K38/47 ,  C07D453/00 ,  C07D453/02 ,  C07D455/02 ,  C12Y302/01022

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

公开(公告)号：US20140255381A1

公开(公告)日：2014-09-11

申请号：US14030725

申请日：2013-09-18

Applicant: GENZYME CORPORATION

Inventor： Elyse BOURQUE ,  Cassandra CELATKA ,  Bradford HIRTH ,  Markus METZ ,  Zhong ZHAO ,  Renato SKLERJ ,  Yibin XIANG ,  Katherine JANCSICS ,  John MARSHALL ,  Seng CHENG ,  Ronald SCHEULE ,  Mario CABRERA-SALAZAR ,  Andrew GOOD

IPC: C07D453/02 ,  A61K38/47 ,  A61K31/439

CPC classification number: C07D487/08 ,  A61K31/439 ,  A61K31/454 ,  A61K31/46 ,  A61K31/506 ,  A61K31/551 ,  A61K38/47 ,  C07D453/00 ,  C07D453/02 ,  C07D455/02 ,  C12Y302/01022

Abstract: The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, 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）抑制剂和癌症治疗。