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：US10913956B2

公开(公告)日：2021-02-09

申请号：US15201268

申请日：2016-07-01

Applicant: Genzyme Corporation

Inventor： Marco A. Passini ,  Greg Stewart ,  James C. Dodge

IPC: A61K48/00 ,  C12N15/86 ,  A61K38/46 ,  A61K38/48

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.