Invention Grant
- Patent Title: Intrathecal administration of adeno-associated-viral vectors for gene therapy
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Application No.: US15771481Application Date: 2016-10-27
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Publication No.: US11135313B2Publication Date: 2021-10-05
- Inventor: James M. Wilson , Christian Hinderer , William Thomas Rothwell
- Applicant: The Trustees of the University of Pennsylvania
- Applicant Address: US PA Philadelphia
- Assignee: The Trustees of the University of Pennsylvania
- Current Assignee: The Trustees of the University of Pennsylvania
- Current Assignee Address: US PA Philadelphia
- Agency: Howson & Howson LLP
- Agent Cathy A. Kodroff
- International Application: PCT/US2016/058968 WO 20161027
- International Announcement: WO2017/075119 WO 20170504
- Main IPC: A61K39/00
- IPC: A61K39/00 ; A61K39/395 ; A61K48/00 ; A61P25/28 ; C07K16/18 ; C12N15/63 ; C12N15/86 ; C07K16/10 ; C12N7/00

Abstract:
A composition comprising at least one AAV vector formulated for intrathecal delivery to the central nervous system is described. The composition comprises at least one expression cassette which contains sequences encoding an immunoglobulin construct linked to expression control sequences therefor and a pharmaceutically acceptable carrier. The immunoglobulin construct may be an immunoglobulin modified to have decreased or no measurable affinity for neonatal Fc receptor (FcRn).
Public/Granted literature
- US20180339065A1 INTRATHECAL ADMINISTRATION OF ADENO-ASSOCIATED-VIRAL VECTORS FOR GENE THERAPY Public/Granted day:2018-11-29
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