	US 12,201,657 B2
	Adeno-associated virus compositions for restoring HBB gene function and methods of use thereof
Saswati Chatterjee, Altadena, CA (US); Kamehameha K. Wong, Jr., Altadena, CA (US); Marwa BenHajSalah, Duarte, CA (US); Laura Jane Smith, Westford, MA (US); Albert Barnes Seymour, Westborough, MA (US); Jason Boke Wright, Concord, MA (US); James Anthony McSwiggen, Arlington, MA (US); Serena Nicole Dollive, Waltham, MA (US); Thia Baboval St. Martin, Lunenburg, MA (US); and Jaime Michelle Prout, Hudson, NH (US)
Assigned to CITY OF HOPE, Duarte, CA (US)
Filed by CITY OF HOPE, Duarte, CA (US)
Filed on Oct. 17, 2018, as Appl. No. 16/163,061.
Claims priority of provisional application 62/621,102, filed on Jan. 24, 2018.
Claims priority of provisional application 62/574,163, filed on Oct. 18, 2017.
Prior Publication US 2019/0134118 A1, May 9, 2019
Int. Cl. A61K 35/761 (2015.01); A61K 48/00 (2006.01); C07K 14/075 (2006.01); C07K 14/805 (2006.01); C12N 15/62 (2006.01); C12N 15/86 (2006.01); C12N 15/90 (2006.01)

CPC A61K 35/761 (2013.01) [A61K 48/0016 (2013.01); A61K 48/005 (2013.01); A61K 48/0058 (2013.01); C07K 14/075 (2013.01); C07K 14/805 (2013.01); C12N 15/62 (2013.01); C12N 15/86 (2013.01); C12N 15/907 (2013.01); A01K 2207/12 (2013.01); A01K 2227/105 (2013.01); A01K 2267/0381 (2013.01)]

3 Claims

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 as set forth in SEQ ID NO: 18;

(ii) a 5′ homology arm nucleotide sequence;

(iii) an editing element for editing a target locus in an HBB gene;

(iv) a 3′ homology arm nucleotide sequence;

wherein the 5′ homology arm, the editing element and the 3′ homology arm of (ii), (iii) and (iv) consists of the nucleotide sequence as set forth in SEQ ID NO: 34 and

(v) a 3′ AAV ITR nucleotide sequence as set forth in SEQ ID NO: 19.