Back to results
US 2022/0333138 A1Published application
Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
AssigneesCity of Hope, Homology Medicines, Inc.
InventorsSaswati CHATTERJEE, Laura Jane SMITH, Jeff Lynn ELLSWORTH, Hillard RUBIN, Jason Boke WRIGHT, James Anthony MCSWIGGEN
Life of this patent
Estimating the term…
Abstract
Provided herein are adeno-associated virus (AAV) compositions that can restore F8 gene function in cell. Also provide are packaging systems for making the adeno-associated virus compositions.
Claims34
1-53. (canceled)
A replication-defective adeno-associated virus (AAV) comprising:
- a) an AAV capsid comprising an AAV Clade F capsid protein;
- and
- b) a correction genome comprising:
- (i) an editing element for editing a target locus in the F8 gene;
- (ii) a 5′ homology arm nucleotide sequence 5′ to the editing element having homology to a first genomic region 5′ to the target locus;
- and (iii) a 3′ homology arm nucleotide sequence 3′ to the editing element having homology to a second genomic region 3′ to the target locus.
3 more elements
56-58. (canceled)
60-70. (canceled)
72-80. (canceled)
82-83. (canceled)
(canceled)
(canceled)
89-91. (canceled)
93-95. (canceled)
97-99. (canceled)
104-105. (canceled)
107-110. (canceled)
112-115. (canceled)
Dependencies are inferred from the claim text.