US 12,419,969 B2
AAV capsid variants and uses thereof
Mathieu Emmanuel Nonnenmacher, Boston, MA (US); Tyler Christopher Moyer, Boston, MA (US); Jiangyu Li, Watertown, MA (US); and Dan Richard Laks, Cambridge, MA (US)
Assigned to VOYAGER THERAPEUTICS, INC., Lexington, MA (US)
Filed by VOYAGER THERAPEUTICS, INC., Lexington, MA (US)
Filed on Apr. 1, 2025, as Appl. No. 19/097,114.
Application 19/097,114 is a division of application No. 18/757,141, filed on Jun. 27, 2024, granted, now 12,296,025.
Application 18/757,141 is a continuation of application No. 18/703,166, previously published as PCT/US2022/079060, filed on Nov. 1, 2022.
Claims priority of provisional application 63/339,711, filed on May 9, 2022.
Claims priority of provisional application 63/274,806, filed on Nov. 2, 2021.
Prior Publication US 2025/0228967 A1, Jul. 17, 2025
Int. Cl. A61K 47/64 (2017.01); A61K 9/00 (2006.01); A61K 48/00 (2006.01); C07K 7/06 (2006.01); C07K 14/005 (2006.01); C12N 15/86 (2006.01)
CPC A61K 48/0041 (2013.01) [A61K 9/0019 (2013.01); A61K 47/64 (2017.08); A61K 48/0058 (2013.01); A61K 48/0075 (2013.01); C07K 7/06 (2013.01); C07K 14/005 (2013.01); C12N 15/86 (2013.01); C12N 2750/14122 (2013.01); C12N 2750/14141 (2013.01); C12N 2830/50 (2013.01)] 30 Claims
 
1. An adeno-associated virus (AAV) capsid variant comprising an amino acid sequence at least 95% identical to amino acids 203-742 of SEQ ID NO: 981, wherein the amino acid sequence comprises SPHSKA (SEQ ID NO: 941) in variable region IV (VR-IV).
 
19. An adeno-associated virus (AAV) capsid variant comprising the amino acid sequence of SEQ ID NO: 981.
 
22. A polynucleotide encoding an adeno-associated virus (AAV) capsid variant comprising an amino acid sequence at least 95% identical to amino acids 203-742 of SEQ ID NO: 981, wherein the amino acid sequence comprises SPHSKA (SEQ ID NO: 941) in variable region IV (VR-IV).
 
25. An adeno-associated virus (AAV) particle comprising an AAV capsid variant comprising an amino acid sequence at least 95% identical to amino acids 203-742 of SEQ ID NO: 981, wherein the amino acid sequence comprises SPHSKA (SEQ ID NO: 941) in variable region IV (VR-IV).