公开(公告)号：US20230372313A1

公开(公告)日：2023-11-23

申请号：US18007433

申请日：2021-07-30

Applicant: GENZYME CORPORATION

Inventor： Tanya Zaremba FISCHER ,  Judith PETERSCHMITT

IPC: A61K31/439 ,  A61P25/16

CPC classification number: A61K31/439 ,  A61P25/16

Abstract: Provided are methods for treating or preventing Parkinson's disease or dementia with Lewy Bodies in a human subject. The methods target lipid dysregulation in brain tissue of the subject and can, in particular, reduce the concentration of glycosphingolipids in brain tissue. The methods use quinuclidine compounds of formula (I), or pharmaceutically acceptable prodrugs or salts thereof. In particular, the human subject may be a carrier of one or more glucocerebrosidase 1 gene (GBA1) mutations, e.g. a heterozygous carrier of GBA1 mutations. Also provided are methods for assessing the effectiveness of treatments as described herein.

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：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.