US 11,840,704 B2
Adeno-associated virus variant capsids and methods of use thereof
David H. Kirn, Emeryville, CA (US); Melissa Kotterman, Emeryville, CA (US); and David Schaffer, Emeryville, CA (US)
Assigned to 4D Molecular Therapeutics Inc., Emeryville, CA (US)
Filed by 4D MOLECULAR THERAPEUTICS INC., Emeryville, CA (US)
Filed on Dec. 21, 2022, as Appl. No. 18/069,701.
Application 18/069,701 is a continuation of application No. 17/718,870, filed on Apr. 12, 2022, granted, now 11,613,766.
Application 17/718,870 is a continuation of application No. 17/400,041, filed on Aug. 11, 2021.
Application 17/400,041 is a continuation of application No. 16/648,195, granted, now 11,118,192, issued on Sep. 14, 2021, previously published as PCT/US2018/051812, filed on Sep. 19, 2018.
Claims priority of provisional application 62/560,901, filed on Sep. 20, 2017.
Prior Publication US 2023/0279435 A1, Sep. 7, 2023
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/86 (2006.01); C07K 14/005 (2006.01); C12N 15/864 (2006.01); A61P 21/00 (2006.01); A61K 38/47 (2006.01); C12N 7/00 (2006.01); A61K 38/17 (2006.01); A61K 38/44 (2006.01); C12N 9/10 (2006.01); A61K 9/00 (2006.01); A61K 48/00 (2006.01)
CPC C12N 15/86 (2013.01) [A61K 38/1719 (2013.01); A61K 38/44 (2013.01); A61K 38/47 (2013.01); A61P 21/00 (2018.01); C07K 14/005 (2013.01); C12N 7/00 (2013.01); C12N 9/1051 (2013.01); C12N 15/8645 (2013.01); C12Y 116/03001 (2013.01); C12Y 204/01001 (2013.01); C12Y 302/0102 (2013.01); C12Y 302/01022 (2013.01); A61K 9/0019 (2013.01); A61K 48/00 (2013.01); C12N 2750/14121 (2013.01); C12N 2750/14122 (2013.01); C12N 2750/14133 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14145 (2013.01); C12N 2750/14171 (2013.01)] 20 Claims
 
1. A recombinant adeno-associated virus (rAAV) comprising:
(i) a variant AAV capsid protein comprising a heterologous peptide insertion with a length of 7 to 20 amino acids covalently inserted in the GH-loop of the capsid protein relative to a corresponding parental AAV capsid protein, wherein the peptide insertion comprises the amino acid sequence NKIQRTD (SEQ ID NO: 13) and
(ii) a heterologous nucleic acid comprising a nucleotide sequence encoding a gene product.