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. 03, 2024
Filed:
May. 27, 2021
Applicant:
Homology Medicines, Inc., Bedford, MA (US);
Inventors:
Jason Boke Wright, Bedford, MA (US);
Danielle Lauren Sookiasian, Bedford, MA (US);
Thia Baboval St. Martin, Bedford, MA (US);
Omar Francone, Bedford, MA (US);
Albert Barnes Seymour, Bedford, MA (US);
Assignee:
Homology Medicines, Inc., Bedford, MA (US);
Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/864 (2006.01); A61K 38/44 (2006.01); A61K 48/00 (2006.01); C12N 15/12 (2006.01); C12N 15/86 (2006.01); C12N 15/90 (2006.01);
U.S. Cl.
CPC ...
A61K 48/0066 (2013.01); A61K 38/44 (2013.01); C12N 15/86 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14151 (2013.01); C12N 2830/50 (2013.01); C12Y 114/16001 (2013.01);
Abstract
Provided herein are recombinant adeno-associated virus (rAAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cells, and methods for using the same to treat diseases associated with reduction of PAH gene function (e.g., PKU). Also provided are nucleic acids, vectors, packaging systems, and methods for making the adeno-associated virus compositions.