公开(公告)号：US11957765B2

公开(公告)日：2024-04-16

申请号：US16808206

申请日：2020-03-03

Applicant: Genzyme Corporation

Inventor： Marco A. Passini ,  James Dodge

IPC: A61K48/00 ,  C07K14/47 ,  C12N15/86 ,  C12N15/864

CPC classification number: A61K48/0075 ,  A61K48/00 ,  C07K14/47 ,  C12N15/86 ,  C12N15/8645 ,  C12N2750/14041 ,  C12N2750/14143 ,  C12N2799/025

Abstract: The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.

公开(公告)号：US20230364200A1

公开(公告)日：2023-11-16

申请号：US18066149

申请日：2022-12-14

Applicant: Genzyme Corporation

Inventor： James Dodge ,  Lamya Shihabuddin ,  Catherine O’Riordan

IPC: A61K38/30 ,  A61K48/00

CPC classification number: A61K38/30 ,  A61K48/005 ,  A61K48/0075 ,  C12N15/8645 ,  C12N2799/025

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.

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：US11554161B2

公开(公告)日：2023-01-17

申请号：US16546070

申请日：2019-08-20

Applicant: Genzyme Corporation

Inventor： James Dodge ,  Lamya Shihabuddin ,  Catherine O'Riordan

IPC: A61K48/00 ,  C12N15/79 ,  C07H21/04 ,  A61K38/30 ,  C12N15/864

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.

公开(公告)号：US11369693B2

公开(公告)日：2022-06-28

申请号：US14328621

申请日：2014-07-10

Applicant: Genzyme Corporation

Inventor： James Dodge ,  Seng Cheng

IPC: A61K48/00 ,  C12N15/79 ,  C07H21/04 ,  C12N15/86 ,  A61K38/47

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.