公开(公告)号：US20170087212A1

公开(公告)日：2017-03-30

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

CPC classification number: A61K38/1709 ,  A61K31/7088 ,  A61K48/00 ,  A61K48/0008 ,  A61K48/005 ,  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：US20210228692A1

公开(公告)日：2021-07-29

申请号：US17137060

申请日：2020-12-29

Applicant: Genzyme Corporation

Inventor： James DODGE ,  Marco A. PASSINI ,  Lamya SHIHABUDDIN ,  Seng H. CHENG

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

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：US20180036295A1

公开(公告)日：2018-02-08

申请号：US15556444

申请日：2016-03-09

Applicant: Genzyme Corporation

Inventor： Seng H. CHENG ,  Lamya SHIHABUDDIN ,  Sergio Pablo SARDI

IPC: A61K31/439 ,  A61K9/00 ,  A61K45/06

CPC classification number: A61K31/439 ,  A61K9/0053 ,  A61K31/00 ,  A61K45/06 ,  A61K2300/00

Abstract: This disclosure relates to a method of treating a proteinopathy in a subject, the method comprising administering to the subject an effective amount of a quinuclidine compound. The disclosure also relates to a method of reducing, reversing or preventing the accumulation of protein aggregates in tissue of a subject diagnosed as having a proteinopathy, or being at risk of developing a proteinopathy, the method comprising administering to the subject an effective amount of a quinuclidine compound. Also disclosed is a pharmaceutical composition comprising a quinuclidine compound for use in said methods. The proteinopathy may be a synucleinopathy or a tauopathy, such as Parkinson's disease, Alzheimer's disease or dementia with Lewy bodies.

公开(公告)号：US20200080066A1

公开(公告)日：2020-03-12

申请号：US16544735

申请日：2019-08-19

Applicant: Genzyme Corporation

Inventor： Marco A. PASSINI ,  Robin J. ZIEGLER ,  James C. DODGE ,  Lamya SHIHABUDDIN ,  Seng H. CHENG

IPC: C12N9/16 ,  C12N15/86 ,  A61K48/00 ,  C12N15/85

Abstract: This disclosure pertains to methods and compositions for tolerizing a mammal's brain to exogenously administered acid sphingomyelinase polypeptide by first delivering an effective amount of a transgene encoding the polypeptide to the mammal's hepatic tissue and then administering an effective amount of the transgene to the mammal's central nervous system (CNS).

公开(公告)号：US20190083582A1

公开(公告)日：2019-03-21

申请号：US16111009

申请日：2018-08-23

Applicant: Genzyme Corporation

Inventor： James DODGE ,  Marco A. PASSINI ,  Lamya SHIHABUDDIN ,  Seng H. CHENG

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

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：US20230287366A1

公开(公告)日：2023-09-14

申请号：US18119201

申请日：2023-03-08

Applicant: Genzyme Corporation

Inventor： Marco A. PASSINI ,  Robin J. ZIEGLER ,  James C. DODGE ,  Lamya SHIHABUDDIN ,  Seng H. CHENG

IPC: C12N9/16 ,  C12N15/85 ,  A61K48/00 ,  C12N15/86

CPC classification number: C12N9/16 ,  C12N15/8509 ,  A61K48/0083 ,  C12N15/86 ,  C12N2840/007 ,  C12N2750/14143 ,  A01K2207/00 ,  A01K2227/105 ,  G01N2500/00 ,  A01K2267/0306

Abstract: This disclosure pertains to methods and compositions for tolerizing a mammal's brain to exogenously administered acid sphingomyelinase polypeptide by first delivering an effective amount of a transgene encoding the polypeptide to the mammal's hepatic tissue and then administering an effective amount of the transgene to the mammal's central nervous system (CNS).

公开(公告)号：US20190111157A1

公开(公告)日：2019-04-18

申请号：US15549962

申请日：2016-02-09

Applicant: Genzyme Corporation

Inventor： Lisa M. STANEK ,  Lamya SHIHABUDDIN

IPC: A61K48/00 ,  A61K9/00 ,  C12N7/00

Abstract: Provided herein are novel methods for delivering recombinant adeno-associated viral (rAAV) particles to the central nervous system of a mammal (e.g., a human). In aspects, the methods involve administering rAAV particles containing a heterologous nucleic acid to the striatum and causing expression of the heterologous nucleic acid in at least the cerebral cortex and the striatum of the mammal.

公开(公告)号：US20160067312A1

公开(公告)日：2016-03-10

申请号：US14692469

申请日：2015-04-21

Applicant: GENZYME CORPORATION

Inventor： James DODGE ,  Lamya SHIHABUDDIN ,  Catherine O'RIORDAN

IPC: A61K38/30

Abstract: This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord 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's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.

Abstract translation: 本公开提供了用于治疗影响受试者的运动功能和控制的疾病或损伤的方法和组合物。 一方面，本发明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的脊髓。 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，将转基因产物递送至受试者的脊髓的组合物。

公开(公告)号：US20160051699A1

公开(公告)日：2016-02-25

申请号：US14876683

申请日：2015-10-06

Applicant: GENZYME CORPORATION

Inventor： Marco A. PASSINI ,  Lamya SHIHABUDDIN ,  Seng H. CHENG

IPC: A61K48/00 ,  A61K38/17 ,  C12N7/00

CPC classification number: A61K38/1709 ,  A61K31/7088 ,  A61K48/00 ,  A61K48/0008 ,  A61K48/005 ,  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：US20150313970A1

公开(公告)日：2015-11-05

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 ,  A61K38/47 ,  A61K9/00

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.