Homology Medicines, Inc.

City of Hope

Jason Boke Wright

Albert Barnes Seymour

Hillard Rubin

Serena Nicole Dollive

Seemin Seher Ahmed

Saswati Chatterjee

Laura Jane Smith

James Anthony Mcswiggen

Jeff Lynn Ellsworth

Danielle Lauren Sookiasian

Jaime Michelle Prout

James A McSwiggen

Kamehameha K Wong, Jr

Omar L Francone

Thia Baboval St Martin

Marwa BenHajSalah

Provided herein are adeno-associated virus (AAV) compositions for correcting a mutation in a beta globin gene (HBB) gene and methods of using the same to correct an HBB gene mutation in a cell. Also provided are packaging systems for making the adeno-associated virus compositions.

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

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

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

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.

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.

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

Provided herein are adeno-associated virus (AAV) compositions that can restore F8 gene function in a cell without co-transducing or co-administering an exogenous nuclease or a nucleotide sequence that encodes an exogenous nuclease. Also provided are methods of using the AAV compositions to correct an F8 gene mutation and/or treat a disease or disorder associated with an F8 gene mutation. Packaging systems for making the adeno-associated virus compositions are also provided.

Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof

Growing community of inventors

Concord, MA, United States of America

Jason Boke Wright