Patent ID: 11891619
Assignee: HOMOLOGY MEDICINES, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC C

Claim 0:
1. 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 from 5′ to 3′:
(i) a 5′ AAV ITR nucleotide sequence;
(ii) a 5′ homology arm nucleotide sequence of up to 2000 nucleotides in length, comprising a nucleotide sequence that is at least 99% identical to SEQ ID NO: 27;
(iii) an editing element for editing a target locus in the F8 gene, comprising an F8 nucleotide sequence that encodes an amino acid sequence consisting of the amino acid sequence encoded by SEQ ID NO: 26 operably linked to a polyadenylation sequence;
(iv) a 3′ homology arm nucleotide sequence of up to 2000 nucleotides in length, comprising a nucleotide sequence that is at least 99% identical to SEQ ID NO: 28; and
(v) a 3′ AAV ITR nucleotide sequence.