The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Sep. 30, 2025
Filed:
Dec. 29, 2022
Applicant:
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Inventors:
James M. Wilson, Philadelphia, PA (US);
Nathan Katz, Stamford, CT (US);
Christian Hinderer, Baltimore, MD (US);
Juliette Hordeaux, Philadelphia, PA (US);
Assignee:
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/86 (2006.01); A61K 48/00 (2006.01); C12N 9/48 (2006.01); A61K 9/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/86 (2013.01); A61K 48/0075 (2013.01); C12N 9/485 (2013.01); A61K 9/0019 (2013.01); C12N 2710/16143 (2013.01); C12N 2750/14143 (2013.01); C12Y 304/14009 (2013.01);
Abstract
Provided herein are methods and compositions for treatment of Batten disease. Such compositions include a recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising (a) an AAV inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human TPP1; (d) an AAV 3' ITR.