Abstract: 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.
Type:
Grant
Filed:
October 19, 2020
Date of Patent:
January 21, 2025
Assignee:
Homology Medicines, Inc.
Inventors:
Albert Barnes Seymour, Seemin Seher Ahmed, Jason Boke Wright, Serena Nicole Dollive, James Anthony McSwiggen, Jaime Michelle Prout, Danielle Lauren Sookiasian
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.
Type:
Grant
Filed:
May 27, 2021
Date of Patent:
September 3, 2024
Assignee:
Homology Medicines, Inc.
Inventors:
Jason Boke Wright, Danielle Lauren Sookiasian, Thia Baboval St. Martin, Omar Francone, Albert Barnes Seymour
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:
February 24, 2020
Date of Patent:
August 20, 2024
Assignee:
Homology Medicines, Inc.
Inventors:
Albert Barnes Seymour, Seemin Seher Ahmed, Jason Boke Wright, Serena Nicole Dollive, Hillard Rubin
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