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.
Type:
Grant
Filed:
March 19, 2020
Date of Patent:
April 9, 2024
Assignee:
HOMOLOGY MEDICINES, INC.
Inventors:
Albert Barnes Seymour, Seemin Seher Ahmed, Jason Boke Wright, Serena Nicole Dollive, Hillard Rubin
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.
Type:
Grant
Filed:
January 13, 2021
Date of Patent:
April 9, 2024
Assignee:
HOMOLOGY MEDICINES, INC.
Inventors:
Omar L. Francone, Deborah A. Kinch, Diana Lamppu, Albert Barnes Seymour, Teresa Leah Wright
Abstract: 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.
Type:
Grant
Filed:
March 10, 2022
Date of Patent:
February 6, 2024
Assignees:
City of Hope, Homology Medicines, Inc.
Inventors:
Saswati Chatterjee, Laura Jane Smith, Jeff Lynn Ellsworth, Hillard Rubin, Jason Boke Wright, James Anthony McSwiggen
Abstract: 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.
Type:
Grant
Filed:
February 19, 2019
Date of Patent:
April 19, 2022
Assignees:
CITY OF HOPE, HOMOLOGY MEDICINES, INC.
Inventors:
Saswati Chatterjee, Laura Jane Smith, Jeff Lynn Ellsworth, Hillard Rubin, Jason Boke Wright, James Anthony McSwiggen
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.
Type:
Grant
Filed:
March 29, 2019
Date of Patent:
April 7, 2020
Assignee:
HOMOLOGY MEDICINES, INC.
Inventors:
Albert Barnes Seymour, Seemin Seher Ahmed, Jason Boke Wright, Serena Nicole Dollive, Hillard Rubin