Invention Grant
- Patent Title: AAV9-mediated gene therapy for treating mucopolysaccharidosis type I
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Application No.: US16624084Application Date: 2018-07-05
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Publication No.: US11890329B2Publication Date: 2024-02-06
- Inventor: Christian Hinderer , James M. Wilson
- 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
- Agent Cathy A. Kodroff
- International Application: PCT/US2018/040957 2018.07.05
- International Announcement: WO2019/010335A 2019.01.10
- Date entered country: 2019-12-18
- Main IPC: A61K38/47
- IPC: A61K38/47 ; A61K9/00 ; A61K31/343 ; A61K31/439 ; A61K35/76 ; A61K45/06 ; C12N7/00 ; C12N9/24 ; C12N15/86 ; A61P3/00 ; A61K48/00
Abstract:
A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene and two or more immunosuppressants is provided herein. Also provided are methods useful for treating hIDUA deficiency (MPSI) and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.
Public/Granted literature
- US20200147185A1 AAV9-MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I Public/Granted day:2020-05-14
Information query
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