公开(公告)号：US11911440B2

公开(公告)日：2024-02-27

申请号：US18054500

申请日：2022-11-10

Applicant: Genzyme Corporation

Inventor： Marco A. Passini ,  Lamya Shihabuddin ,  Seng H. Cheng

IPC: A61K48/00 ,  C12N15/86 ,  A61K38/17 ,  A61K31/7088 ,  C12N7/00 ,  C07K14/47

CPC classification number: A61K38/1709 ,  A61K31/7088 ,  A61K48/00 ,  A61K48/005 ,  A61K48/0008 ,  A61K48/0075 ,  C07K14/4702 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14171

Abstract: Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

公开(公告)号：US10080783B2

公开(公告)日：2018-09-25

申请号：US14559851

申请日：2014-12-03

Applicant: Genzyme Corporation

Inventor： James Dodge ,  Marco A. Passini ,  Lamya Shihabuddin ,  Seng H. Cheng

IPC: A61K38/46 ,  A61K9/00 ,  A61K38/47

CPC classification number: A61K38/465 ,  A61K9/0019 ,  A61K38/47 ,  C12Y301/04012 ,  C12Y301/06013 ,  C12Y302/0102 ,  C12Y302/01045 ,  C12Y302/01076 ,  C12Y304/14

Abstract: Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.

公开(公告)号：US20150210681A1

公开(公告)日：2015-07-30

申请号：US14427275

申请日：2013-09-10

Applicant: GENZYME CORPORATION

Inventor： Elyse Bourque ,  Mario A. Cabrera-Salazar ,  Cassandra Celatka ,  Seng H. Cheng ,  Bradford Hirth ,  Andrew Good ,  Katherine Jancsics ,  John Marshall ,  Markus Metz ,  Ronald K. Scheule ,  Renato Skerlj ,  Yibin Xiang ,  Zhong Zhao ,  John Leonard ,  Thomas Natoli ,  Elina Makino ,  Herve Husson ,  Oxana Beskrovnaya

IPC: C07D453/02 ,  C07D471/08 ,  A61K31/55 ,  A61K45/06 ,  A61K31/506 ,  A61K31/497 ,  A61K31/439 ,  A61K31/5377 ,  A61K31/496 ,  A61K31/551

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：US12060349B2

公开(公告)日：2024-08-13

申请号：US17230385

申请日：2021-04-14

Applicant: Genzyme Corporation

Inventor： Elyse Bourque ,  Mario A. Cabrera-Salazar ,  Cassandra Celatka ,  Seng H. Cheng ,  Bradford Hirth ,  Andrew Good ,  Katherine Jancsics ,  John Marshall ,  Markus Metz ,  Ronald K. Scheule ,  Renato Skerlj ,  Yibin Xiang ,  Zhong Zhao ,  John Leonard ,  Thomas Natoli ,  Elina Makino ,  Herve Husson ,  Oxana Beskrovnaya

IPC: C07D453/02 ,  A61K31/439 ,  A61K31/496 ,  A61K31/497 ,  A61K31/506 ,  A61K31/5377 ,  A61K31/55 ,  A61K31/551 ,  A61K45/06 ,  C07D471/08

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) of the Formula I: represented by the following structural formula




or a pharmaceutically acceptable salt thereof, wherein the variables are as defined herein, 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.

公开(公告)号：US10907142B2

公开(公告)日：2021-02-02

申请号：US16268989

申请日：2019-02-06

Applicant: Genzyme Corporation

Inventor： Yunxiang Zhu ,  Seng H. Cheng ,  Canwen Jiang ,  Luis Z. Avila

IPC: C07H15/04 ,  C12N9/96 ,  C12N9/16 ,  C12N9/26 ,  A61K47/54 ,  A61K38/47

Abstract: The invention provides methods for the synthesis of oligosaccharides comprising an aminooxy group. The invention further provides oligosaccharides comprising an aminooxy group, methods for coupling oligosaccharides comprising an aminooxy group to glycoproteins, and oligosaccharide-protein conjugates. Also provided are methods of treating a lysosomal storage disorder in a mammal by administration of an oligosaccharide-protein conjugate.

公开(公告)号：US10220039B2

公开(公告)日：2019-03-05

申请号：US15414838

申请日：2017-01-25

Applicant: Genzyme Corporation

Inventor： Thomas A. Natoli ,  Oxana Ibraghimov-Beskrovnaya ,  John P. Leonard ,  Nelson S. Yew ,  Seng H. Cheng

IPC: A61K31/5377 ,  A61K31/4439 ,  A61K31/427 ,  A61K31/422 ,  A61K31/421 ,  A61K31/40 ,  A61K31/27 ,  A61K31/4025

Abstract: A method of treating polycystic kidney disease in a subject comprises administering to the subject an effective amount of a compound represented by Structural Formula (1): or a pharmaceutically acceptable salt thereof.

公开(公告)号：US11253485B2

公开(公告)日：2022-02-22

申请号：US16281961

申请日：2019-02-21

Applicant: Genzyme Corporation

Inventor： James C. Dodge ,  Marco A. Passini ,  Lamya S. Shihabuddin ,  Seng H. Cheng

IPC: A61K31/00 ,  A61K9/00

Abstract: Neurological diseases, including lysosomal storage diseases, can be successfully treated using intraventricular delivery of the therapeutic agents to bypass the blood-brain barrier. Similarly, diagnostic agents and anesthetic agents can be delivered to the brain in this manner. The administration can be performed slowly to achieve maximum effect. Such administration permits greater penetration of distal portions of the brain.

公开(公告)号：US11008316B2

公开(公告)日：2021-05-18

申请号：US15490385

申请日：2017-04-18

Applicant: Genzyme Corporation

Inventor： Elyse Bourque ,  Mario A. Cabrera-Salazar ,  Cassandra Celatka ,  Seng H. Cheng ,  Bradford Hirth ,  Andrew Good ,  Katherine Jancsics ,  John Marshall ,  Markus Metz ,  Ronald K. Scheule ,  Renato Skerlj ,  Yibin Xiang ,  Zhong Zhao ,  John Leonard ,  Thomas Natoli ,  Elina Makino ,  Herve Husson ,  Oxana Beskrovnaya

IPC: C07D453/02 ,  C07D471/08 ,  A61K31/439 ,  A61K31/496 ,  A61K31/497 ,  A61K31/506 ,  A61K31/5377 ,  A61K31/55 ,  A61K31/551 ,  A61K45/06

Abstract: The invention relates to inhibitors of glucosylceramide synthase (GCS), such as Compound of Formula I, shown below, as defined herein, 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.

公开(公告)号：US10369193B2

公开(公告)日：2019-08-06

申请号：US15160949

申请日：2016-05-20

Applicant: Genzyme Corporation

Inventor： Marco A. Passini ,  Lamya Shihabuddin ,  Seng H. Cheng

IPC: A61K38/17 ,  C12N7/00 ,  A61K48/00 ,  C07K14/47 ,  A61K31/7088 ,  C12N15/86

Abstract: Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

公开(公告)号：US20170281593A1

公开(公告)日：2017-10-05

申请号：US15383602

申请日：2016-12-19

Applicant: Genzyme Corporation

Inventor： Hongmei Zhao ,  Nelson S. Yew ,  Seng H. Cheng ,  Canwen Jiang ,  Cynthia Marie Arbeeny

IPC: A61K31/395 ,  C07D405/06 ,  A61K31/4025 ,  A61K31/4245 ,  A61K31/40 ,  A61K45/06

CPC classification number: A61K31/395 ,  A61K31/00 ,  A61K31/40 ,  A61K31/4025 ,  A61K31/4245 ,  A61K45/00 ,  A61K45/06 ,  C07D405/06

Abstract: The disclosure provides methods for treating fatty liver disease and associated conditions by inhibiting the synthesis of glucosphingolipids, as exemplified by the use of glucosylceramide synthase substrate analogs.