The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Feb. 06, 2024
Filed:
Mar. 10, 2022
City of Hope, Duarte, CA (US);
Homology Medicines, Inc., Bedford, MA (US);
Saswati Chatterjee, Altadena, CA (US);
Laura Jane Smith, Bedford, MA (US);
Jeff Lynn Ellsworth, Lexington, MA (US);
Hillard Rubin, Northborough, MA (US);
Jason Boke Wright, Bedford, MA (US);
James Anthony McSwiggen, Arlington, MA (US);
City of Hope, Duarte, CA (US);
Homology Medicines, Inc., Bedford, MA (US);
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.