公开(公告)号：US20160166542A1

公开(公告)日：2016-06-16

申请号：US15049946

申请日：2016-02-22

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

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

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.

Abstract translation: 公开了由以下结构式表示的化合物的盐酸盐：（式I Hemitartrate），其可用于药物应用。 式（I）的特殊单晶形式酒石酸盐的特征在于多种性质和物理测量。 此外，还讨论了生产结晶式（I）酒石酸盐并使用其抑制葡萄糖神经酰胺合成酶或降低受试者中的糖鞘醇浓度以治疗许多疾病的方法。 还描述了药物组合物。

公开(公告)号：US09139580B2

公开(公告)日：2015-09-22

申请号：US14030725

申请日：2013-09-18

Applicant: GENZYME CORPORATION

Inventor： Elyse Bourque ,  Bradford Hirth ,  Renato Sklerj ,  Elina Makino ,  Fazeela Morshed ,  Lingyun Li ,  Paul Mason ,  John P. Leonard ,  James Lillie ,  Hanlan Liu ,  Mary A. Cromwell ,  Bing Wang ,  Thomas O'Shea

IPC: A01N43/90 ,  A61K31/44 ,  C07D453/02 ,  A61K38/47 ,  A61K31/439 ,  A61K31/454 ,  C07D453/00

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：US09126993B2

公开(公告)日：2015-09-08

申请号：US14471958

申请日：2014-08-28

Applicant: GENZYME CORPORATION

Inventor： Elyse Bourque ,  Bradford Hirth ,  Renato Sklerj ,  Elina Makino ,  Fazeela Morshed ,  Lingyun Li ,  Paul Mason ,  John P. Leonard ,  James Lillie ,  Hanlan Liu ,  Mary A. Cromwell ,  Bing Wang ,  Thomas O'Shea

IPC: A01N43/90 ,  A61K31/44 ,  C07D453/02 ,  A61K38/47 ,  A61K31/439 ,  A61K31/454 ,  C07D453/00

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。

公开(公告)号：US20160120842A1

公开(公告)日：2016-05-05

申请号：US14994489

申请日：2016-01-13

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

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

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.