公开(公告)号：WO2015179759A1

公开(公告)日：2015-11-26

申请号：PCT/US2015/032170

申请日：2015-05-22

Applicant: GENZYME CORPORATION ,  PAPOULIS, Andrew

Inventor： PAPOULIS, Andrew ,  MASON, Paul ,  CARTER, Kara ,  JOSEPH, Alexandra ,  HU, Yiding ,  GREGORY, Jill ,  ZHAO, Zhong ,  YEE, Christopher ,  MOHAMUD, Mohamud ,  XIANG, Yibin ,  DANTHI, Sanjay ,  HUANG, Yinyin

IPC: C07D491/107 ,  C07D491/20 ,  C07D498/10 ,  A61K31/438 ,  A61K31/444 ,  A61K31/537 ,  A61P31/12 ,  A61P35/00 ,  A61P37/06

CPC classification number: C07D498/10 ,  C07D491/107 ,  C07D491/20 ,  C12N5/0635 ,  G01N33/5008 ,  G01N33/60

Abstract: The invention features compounds of Formula (I), Formula (I)-A and Formula (I)- B as disclosed herein, as well as methods of synthesis, therapy, diagnostics, and assays.

Abstract translation: 本发明的特征在于本文公开的式（I），式（I）-A和式（I）-B的化合物，以及合成，治疗，诊断和测定方法。

公开(公告)号：WO2014043068A1

公开(公告)日：2014-03-20

申请号：PCT/US2013/058896

申请日：2013-09-10

Applicant: GENZYME CORPORATION

Inventor： BOURQUE, Elyse ,  CABRERA-SALAZAR, Mario, A. ,  CELATKA, Cassandra ,  CHENG, Seng, H. ,  HIRTH, Bradford ,  GOOD, Andrew ,  JANCSICS, Katherine ,  MARSHALL, John ,  METZ, Markus ,  SCHEULE, Ronald, K. ,  SKERLJ, Renato ,  XIANG, Yibin ,  ZHAO, Zhong ,  LEONARD, John ,  NATOLI, Thomas ,  MAKINO, Elina ,  HUSSON, Herve ,  BESKROVNAYA, Oxana

IPC: C07D453/02 ,  C07D471/08 ,  A61K31/439 ,  A61P25/00 ,  A61P35/00

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：WO2012129084A3

公开(公告)日：2012-09-27

申请号：PCT/US2012/029417

申请日：2012-03-16

Applicant: GENZYME CORPORATION ,  BOURQUE, Elyse ,  CELATKA, Cassandra ,  HIRTH, Bradford ,  METZ, Markus ,  ZHAO, Zhong ,  SKERLJ, Renato ,  XIANG, Yibin ,  JANCISICS, Katherine ,  MARSHALL, John ,  CHENG, Seng ,  SCHEULE, Ronald ,  CABRERA-SALAZAR, Mario ,  GOOD, Andrew

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

IPC: A01N43/00

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.