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:
Jun. 24, 2025
Filed:
May. 02, 2020
Applicant:
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Inventors:
James M. Wilson, Philadelphia, PA (US);
Juliette Hordeaux, Philadelphia, PA (US);
Assignee:
Attorneys:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C12N 15/86 (2006.01); A61K 47/02 (2006.01); A61K 48/00 (2006.01); A61P 25/28 (2006.01); C12N 9/16 (2006.01);
U.S. Cl.
CPC ...
C12N 15/86 (2013.01); A61K 47/02 (2013.01); A61P 25/28 (2018.01); C12N 9/16 (2013.01); C12Y 301/06001 (2013.01); A61K 48/00 (2013.01); C12N 2750/14121 (2013.01); C12N 2750/14122 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14171 (2013.01); C12N 2830/42 (2013.01); C12N 2830/50 (2013.01);
Abstract
Provided is a recombinant adeno-associated virus (rAAV) having an AAVhu68 capsid and a vector genome which comprises a nucleic acid sequence encoding a functional human arylsulfatase A (ARSA). Also provided are a production system useful for producing the rAAV, a pharmaceutical composition comprising the rAAV, and a method of treating a subject having metachromatic leukodystrophy, or ameliorating symptoms of metachromatic leukodystrophy, or delaying progression of metachromatic leukodystrophy via administering an effective amount of rAAV to a subject in need thereof.