公开(公告)号：US20210261557A1

公开(公告)日：2021-08-26

申请号：US17242016

申请日：2021-04-27

Applicant: GENZYME CORPORATION

Inventor： Elyse BOURQUE ,  Mario CABRERA-SALAZAR ,  Cassandra CELATKA ,  Seng CHENG ,  Mary A. CROMWELL ,  Andrew GOOD ,  Bradford HIRTH ,  Katherine JANCSICS ,  John P. LEONARD ,  Lingyun LI ,  James LILLIE ,  Hanlan LIU ,  Elina MAKINO ,  John MARSHALL ,  Paul MASON ,  Markus METZ ,  Fazeela MORSHED ,  Thomas O'SHEA ,  Ronald SCHEULE ,  Renato SKERLJ ,  Bing WANG ,  Yibin XIANG ,  Zhong ZHAO

IPC: C07D487/08 ,  A61K31/439 ,  A61K31/454 ,  A61K38/47 ,  C07D453/02 ,  C07D453/00 ,  A61K31/46 ,  A61K31/506 ,  A61K31/551 ,  C07D455/02

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, and for the treatment of cancer.

公开(公告)号：US20230172903A1

公开(公告)日：2023-06-08

申请号：US17865195

申请日：2022-07-14

Applicant: Genzyme Corporation

Inventor： Hanlan LIU ,  Chris WILLIS ,  Renu BHARDWAJ ,  Jianmei KOCHLING ,  Judith PETERSCHMITT ,  Craig SIEGEL

IPC: A61K31/4025 ,  C07D405/06 ,  A61K45/06 ,  C07D319/16

CPC classification number: A61K31/4025 ,  C07D405/06 ,  A61K45/06 ,  C07D319/16

Abstract: The hemitartrate salt of a compound represented by the following structural formula:




(Formula I Hemitartrate), which may be used in pharmaceutical applications, are disclosed. Particular single crystalline forms of the Formula (I) Hemitartrate are characterized by a variety of properties and physical measurements. As well, methods of producing crystalline Formula (I) Hemitartrate, and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations in subjects to treat a number of diseases, are also discussed. Pharmaceutical compositions are also described.

公开(公告)号：US20200048266A1

公开(公告)日：2020-02-13

申请号：US16542166

申请日：2019-08-15

Applicant: GENZYME CORPORATION

Inventor： Elyse BOURQUE ,  Mario CABRERA-SALAZAR ,  Cassandra CELATKA ,  Seng CHENG ,  Mary A. CROMWELL ,  Andrew GOOD ,  Bradford HIRTH ,  Katherine JANCSICS ,  John P. LEONARD ,  Lingyun LI ,  James LILLIE ,  Hanlan LIU ,  Elina MAKINO ,  John MARSHALL ,  Paul MASON ,  Markus METZ ,  Fazeela MORSHED ,  Thomas O'SHEA ,  Ronald SCHEULE ,  Renato SKERLJ ,  Bing WANG ,  Yibin XIANG ,  Zhong ZHAO

IPC: C07D487/08 ,  A61K31/439 ,  A61K31/454 ,  A61K38/47 ,  C07D453/02 ,  C07D453/00 ,  A61K31/46 ,  A61K31/506 ,  A61K31/551 ,  C07D455/02

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, and for the treatment of cancer.

公开(公告)号：US20160207933A1

公开(公告)日：2016-07-21

申请号：US14835402

申请日：2015-08-25

Applicant: GENZYME CORPORATION

Inventor： Elyse BOURQUE ,  Mario CABRERA-SALAZAR ,  Cassandra CELATKA ,  Seng CHENG ,  Mary A. CROMWELL ,  Andrew GOOD ,  Bradford HIRTH ,  Katherine JANCSICS ,  John P. LEONARD ,  Lingyun LI ,  James LILLIE ,  Hanlan LIU ,  Elina MAKINO ,  John MARSHALL ,  Paul MASON ,  Markus METZ ,  Fazeela MORSHED ,  Thomas O'SHEA ,  Ronald SCHEULE ,  Renato SKERLJ ,  Bing WANG ,  Yibin XIANG ,  Zhong ZHAO

IPC: C07D487/08 ,  A61K31/439 ,  A61K38/47 ,  A61K31/551 ,  C07D455/02 ,  A61K31/46 ,  C07D453/02 ,  A61K31/506

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。

公开(公告)号：US20210393590A1

公开(公告)日：2021-12-23

申请号：US17465994

申请日：2021-09-03

Applicant: Genzyme Corporation

Inventor： Hanlan LIU ,  Chris WILLIS ,  Renu BHARDWAJ ,  Diane P. COPELAND ,  Abizer HARIANAWALA ,  Jeffrey SKELL ,  John MARSHALL ,  Jianmei KOCHLING ,  Gerard PALACE ,  Judith PETERSCHMITT ,  Craig SIEGEL ,  Seng CHENG

IPC: A61K31/4025 ,  C07D405/06 ,  C07D319/16 ,  A61K45/06

Abstract: The hemitartrate salt of a compound represented by the following structural formula: (Formula 1 Hemitartrate), which may be used in pharmaceutical applications, are disclosed. Particular single crystalline forms of the Formula (I) Hemitartrate are characterized by a variety of properties and physical measurements. As well, methods of producing crystalline Formula (I) Hemitartrate, and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations in subjects to treat a number of diseases, are also discussed. Pharmaceutical compositions are also described.

公开(公告)号：US20210369672A1

公开(公告)日：2021-12-02

申请号：US17143821

申请日：2021-01-07

Applicant: Genzyme Corporation

Inventor： Hanlan LIU ,  Chris WILLIS ,  Renu BHARDWAJ ,  Diane P. COPELAND ,  Abizer HARIANAWALA ,  Jeffrey SKELL ,  John MARSHALL ,  Jianmei KOCHLING ,  Gerard PALACE ,  Judith PETERSCHMITT ,  Craig SIEGEL ,  Seng CHENG

IPC: A61K31/4025 ,  C07D405/06 ,  A61K45/06 ,  C07D319/16

Abstract: The hemitartrate salt of a compound represented by the following structural formula: (Formula I Hemitartrate), which may be used in pharmaceutical applications, are disclosed. Particular single crystalline forms of the Formula (I) Hemitartrate are characterized by a variety of properties and physical measurements. As well, methods of producing crystalline Formula (I) Hemitartrate, and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations in subjects to treat a number of diseases, are also discussed. Pharmaceutical compositions are also described.