Patent · US Active

Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof

US12076420B2 · kind B2 · utility

0Cited by

84References

18Claims

0Family size

Assignee

Homology Medicines, Inc. · US

Inventors

Jason Boke Wright · Concord, US
Danielle Lauren Sookiasian · Quincy, US
Thia Baboval St. Martin · Lunenburg, US
Omar Francone · Bedford, US
Albert Barnes Seymour · Westborough, US

Key dates

Filing date	May 27, 2021
Grant date	Sep 3, 2024
Priority date	—
Expiry date	Oct 16, 2041

Classification

Technology area (CPC C)Chemistry; Metallurgy
CPC primaryC12Y114/16001
WIPO fieldPharmaceuticals
WIPO sectorChemistry

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.

Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.