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:
Apr. 09, 2024
Filed:
Jan. 13, 2021
Applicant:
Homology Medicines, Inc., Bedford, MA (US);
Inventors:
Omar L. Francone, Waltham, MA (US);
Deborah A. Kinch, Bedford, MA (US);
Diana Lamppu, Roslindale, MA (US);
Albert Barnes Seymour, Westborough, MA (US);
Teresa Leah Wright, Lexington, MA (US);
Assignee:
HOMOLOGY MEDICINES, INC., Bedford, MA (US);
Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/864 (2006.01); A61K 48/00 (2006.01); C12N 7/00 (2006.01); C12N 7/01 (2006.01); C12N 9/02 (2006.01); C12N 15/52 (2006.01); C12N 15/86 (2006.01); A61K 31/573 (2006.01);
U.S. Cl.
CPC ...
C12N 15/86 (2013.01); C12N 7/00 (2013.01); C12N 9/0071 (2013.01); A61K 31/573 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01); C12N 2830/50 (2013.01); C12Y 114/16001 (2013.01);
Abstract
Provided herein are methods of treating a subject having a disease or disorder associated with a PAH gene mutation. The methods generally comprise administering to the subject a therapeutically effective does of a recombinant adeno-associated virus (rAAV) that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell and thereby restore PAH gene function in the subject.