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。

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：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：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.

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：US20150151007A1

公开(公告)日：2015-06-04

申请号：US14328621

申请日：2014-07-10

Applicant: Genzyme Corporation

Inventor： James DODGE ,  Seng CHENG

IPC: A61K48/00 ,  A61K38/47

CPC classification number: A61K48/0075 ,  A61K38/47 ,  C12N15/86 ,  C12N2750/14143 ,  C12Y302/01045

Abstract: This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.

Abstract translation: 本公开提供了用于治疗受试者中溶酶体贮积病的方法和组合物。 在本发明的一个方面，通过将包含转基因的重组神经营养病毒载体给予脑，将转基因产物递送至受试者。 病毒载体将转基因递送到易受病毒感染并表达编码的重组病毒基因产物的脑区域。 还提供了通过将含有转基因的重组神经营养病毒载体给予受试者的脑，将转基因产物递送给受试者的组合物。 转基因产物可以是溶酶体贮积病中缺乏的任何一种。