Invention Grant
- Patent Title: Gene therapy for treating familial hypercholesterolemia
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Application No.: US16060409Application Date: 2016-12-09
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Publication No.: US10889832B2Publication Date: 2021-01-12
- Inventor: James M. Wilson , Daniel J. Rader
- 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 and Howson LLP
- Agent Cathy A. Kodroff
- International Application: PCT/US2016/065984 WO 20161209
- International Announcement: WO2017/100682 WO 20170615
- Main IPC: A61K48/00
- IPC: A61K48/00 ; C12N15/861 ; C07K14/705 ; C12N15/86 ; A61P3/06 ; A61K9/00

Abstract:
Compositions and regimens useful in reducing one or more of: LDL-cholesterol, total cholesterol, and/or fasting triglycerides in a subject, and/or modifying fractional catabolic rate (FCR) of LDL apolipoprotein B (apoB) from baseline to a selected time point after rAAV administration are provided. The method involves administering to the human subject via a peripheral vein by infusion of a suspension of replication deficient recombinant adeno-associated virus (rAAV).
Public/Granted literature
- US20190002917A1 GENE THERAPY FOR TREATING FAMILIAL HYPERCHOLESTEROLEMIA Public/Granted day:2019-01-03
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