Invention Grant
- Patent Title: Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
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Application No.: US17654300Application Date: 2022-03-10
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Publication No.: US11891619B2Publication Date: 2024-02-06
- Inventor: Saswati Chatterjee , Laura Jane Smith , Jeff Lynn Ellsworth , Hillard Rubin , Jason Boke Wright , James Anthony McSwiggen
- Applicant: City of Hope , Homology Medicines, Inc.
- Applicant Address: US CA Duarte
- Assignee: City of Hope,Homology Medicines, Inc.
- Current Assignee: City of Hope,Homology Medicines, Inc.
- Current Assignee Address: US CA Duarte; US MA Bedford
- Agency: Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C.
- The original application number of the division: US16279688 2019.02.19
- Main IPC: C12N15/86
- IPC: C12N15/86 ; C12N15/90 ; C12N7/00
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.
Public/Granted literature
- US20220333138A1 ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING F8 GENE FUNCTION AND METHODS OF USE THEREOF Public/Granted day:2022-10-20
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