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:
Jan. 17, 2023
Filed:
Nov. 29, 2018
Applicant:
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Inventors:
James M. Wilson, Philadelphia, PA (US);
Nathan Katz, Stamford, CT (US);
Juliette Hordeaux, Philadelphia, PA (US);
Assignee:
Other;
Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/86 (2006.01); A61P 19/00 (2006.01); A61K 35/76 (2015.01); A61K 48/00 (2006.01); C12N 7/00 (2006.01); C12N 9/14 (2006.01);
U.S. Cl.
CPC ...
C12N 15/86 (2013.01); A61K 35/76 (2013.01); A61K 48/005 (2013.01); A61P 19/00 (2018.01); C12N 7/00 (2013.01); C12N 9/14 (2013.01); C12Y 310/01001 (2013.01); C12N 2750/14122 (2013.01); C12N 2750/14143 (2013.01);
Abstract
Provided herein is a recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5' inverted terminal repeat (ITR), an engineered nucleic acid sequence encoding a functional hSGSH, a regulatory sequence which direct expression of hSGSH in a target cell, and an AAV 3′ ITR. Also provided is a pharmaceutical composition comprising a rAAV as described herein in a formulation buffer, and a method of treating a human subject diagnosed with MPS IIIA.