US 12,281,321 B2
Frataxin expression constructs having engineered promoters and methods of use thereof
Holger Patzke, Lexington, MA (US); Jinzhao Hou, Lexington, MA (US); Hongxing Wang, Lexington, MA (US); Yanqun Shu, Lexington, MA (US); Martin Goulet, Lexington, MA (US); and Dinah Wen-Yee Sah, Lexington, MA (US)
Assigned to Voyager Therapeutics, Inc., Lexington, MA (US)
Appl. No. 17/279,878
Filed by VOYAGER THERAPEUTICS, INC., Lexington, MA (US)
PCT Filed Sep. 27, 2019, PCT No. PCT/US2019/053681
§ 371(c)(1), (2) Date Mar. 25, 2021,
PCT Pub. No. WO2020/069461, PCT Pub. Date Apr. 2, 2020.
Claims priority of provisional application 62/901,769, filed on Sep. 17, 2019.
Claims priority of provisional application 62/738,519, filed on Sep. 28, 2018.
Prior Publication US 2021/0395776 A1, Dec. 23, 2021
Int. Cl. C12N 15/86 (2006.01); A61K 47/02 (2006.01); A61K 47/10 (2017.01); A61K 48/00 (2006.01); C07K 14/47 (2006.01)
CPC C12N 15/86 (2013.01) [A61K 47/02 (2013.01); A61K 47/10 (2013.01); A61K 48/005 (2013.01); C07K 14/47 (2013.01); C12N 2830/008 (2013.01)] 42 Claims
 
1. An adeno-associated viral (AAV) vector genome comprising an engineered promoter and a nucleic acid sequence encoding a frataxin protein, wherein the engineered promoter consists of a sequence that has at least 90% sequence identity to SEQ ID NO: 1742 over the full length of the engineered promoter.