Patent · US Active

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

US12064486B2 · kind B2 · utility

0Cited by

80References

20Claims

0Family size

Assignee

Homology Medicines, Inc. · US

Inventors

Albert Barnes Seymour · Westborough, US
Seemin Seher Ahmed · Worcester, US
Jason Boke Wright · Concord, US
Serena Nicole Dollive · Waltham, US
Hillard Rubin · Northborough, US

Key dates

Filing date	Feb 24, 2020
Grant date	Aug 20, 2024
Priority date	—
Expiry date	Oct 17, 2042

Classification

Technology area (CPC C)Chemistry; Metallurgy
CPC primaryC12N2830/32
WIPO fieldPharmaceuticals
WIPO sectorChemistry

Abstract

Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.

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