US 11,752,181 B2
Compositions and methods of treating Huntington's disease
Dinah Wen-Yee Sah, Hopkinton, MA (US); Fen Chen, Saugus, MA (US); Pengcheng Zhou, Lexington, MA (US); Xin Wang, Arlington, MA (US); Yanqun Shu, Winchester, MA (US); Jinzhao Hou, Lexington, MA (US); Jochen Deckert, Bayreuth (DE); and Markus Hossbach, Kulmbach (DE)
Assigned to VOYAGER THERAPEUTICS, INC., Cambridge, MA (US)
Appl. No. 16/611,041
Filed by VOYAGER THERAPEUTICS, INC., Cambridge, MA (US)
PCT Filed May 4, 2018, PCT No. PCT/US2018/031117
§ 371(c)(1), (2) Date Nov. 5, 2019,
PCT Pub. No. WO2018/204803, PCT Pub. Date Nov. 8, 2018.
Claims priority of provisional application 62/520,086, filed on Jun. 15, 2017.
Claims priority of provisional application 62/507,921, filed on May 18, 2017.
Claims priority of provisional application 62/501,786, filed on May 5, 2017.
Prior Publication US 2020/0155624 A1, May 21, 2020
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 35/76 (2015.01); C12N 15/113 (2010.01); A61P 25/14 (2006.01); C12N 7/00 (2006.01); C12N 15/86 (2006.01); A61K 48/00 (2006.01)
CPC A61K 35/76 (2013.01) [A61P 25/14 (2018.01); C12N 7/00 (2013.01); C12N 15/113 (2013.01); C12N 15/86 (2013.01); A61K 48/00 (2013.01); C12N 2310/141 (2013.01); C12N 2320/32 (2013.01); C12N 2750/14121 (2013.01); C12N 2750/14123 (2013.01); C12N 2750/14132 (2013.01); C12N 2750/14143 (2013.01)] 21 Claims
 
1. An adeno-associated viral (AAV) genome comprising a first inverted terminal repeat (ITR), a promoter operably linked to a nucleic acid comprising a modulatory polynucleotide encoding a sense strand sequence and an antisense strand sequence, and a second ITR; wherein the encoded antisense strand sequence comprises the nucleotide sequence of SEQ ID NO: 918 and the sense strand sequence comprises the nucleotide sequence of SEQ ID NO: 1079; and wherein the AAV viral genome comprises one or more of:
(i) a first ITR sequence comprising the nucleotide sequence of any one of SEQ ID NOs: 1380 or 1381-1383;
(ii) a promoter comprising a chicken β-actin (CBA) promoter, a cytomegalovirus (CMV) promoter, a phosphoglycerate kinase 1 (PGK) promoter, an H1 promoter, a T7 promoter, a ubiquitin c (UBC) promoter, a β-glucuronidase (GUSB) promoter, a neuron-specific enolase (NSE) promoter, a synapsin promoter, a methyl-CpG binding protein 2 (MeCP2) promoter, or a glial fibrillary acidic protein (GFAP) promoter;
(iii) a promoter comprising the nucleotide sequence of any one of SEQ ID NOs: 1410, 1411-1414; or
(iv) a second ITR sequence comprising the nucleotide sequence of any one of SEQ ID NOs: 1382, 1380, 1381, or 1383.