IP Library › Granted Patent US 10,912,804
Granted Patent B2
US 10,912,804 · App. 15/088,167 · Granted Feb 9, 2021

Compositions and methods for treating diseases

Inventors: Barry John Byrne (Gainesville, FL); Darin J. Falk (Gainesville, FL); Christina Pacak (Gainesville, FL); Lara Robert DeRuisseau (Syracuse, NY); Cathryn Mah (Irvine, CA); David D. Fuller (Gainesville, FL)
Assignee: University of Florida Research Foundation, Incorporated
A61K35/76A61K9/0019A61K48/005C12N7/00C12N9/2408C12N15/86C12Y302/0102C12N2710/16644C12N2750/14111C12N2750/14143C12N2750/14152
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Quick Facts
Patent No.
US 10,912,804
App. No.
15/088,167
Granted
Feb 9, 2021
Kind
B2
Abstract

The present invention provides compositions and methods of use pertaining to rAAV-mediated delivery of therapeutically effective molecules for treatment of diseases such as Pompe disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells.

Claims (3)

1. A method of treating Pompe disease in a human patient suffering therefrom, said method comprising administering to said patient a therapeutically effective amount of a pseudotyped recombinant adeno-associated virus (rAAV) selected from the group consisting of: rAAV2/8 and rAAV2/9, wherein said rAAV comprises a nucleic acid encoding a human alpha-glucosidase (GAA) polypeptide operably linked to a desmin promoter, wherein said rAAV is administered to said patient intravenously, intrathecally, intraspinally, and/or intramuscularly.

2. The method of claim 1 , wherein said rAAV is rAAV2/9.

3. The method of claim 1 , wherein said rAAV is rAAV2/8.

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Dec 23, 2016
From: BYRNE, BARRY JOHN; FALK, DARIN J.; PACAK, CHRISTINA; DERUISSEAU, LARA ROBERT; MAH, CATHRYN; FULLER, DAVID D.
To: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED
Reel/Frame 040759/0598 →
CONFIRMATORY LICENSE Recorded Oct 3, 2016
From: UNIVERSITY OF FLORIDA
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 040204/0413 →
Continuity (5)
Continuation 14409643
Continuation In Part 13527350 · Jun 19, 2012
Continuation In Part 12305869
Provisional Application 60891369 · Feb 23, 2007
Related Publication 20170028002A1 · Feb 2, 2017
Cited By (3)
US 12,215,138 US 12,252,544 US 12,258,597