US 11,931,375 B2
Treatment of amyotrophic lateral sclerosis (ALS)
Dinah Wen-Yee Sah, Hopkinton, MA (US); Holger Patzke, Cambridge, MA (US); Qingmin Chen, Belmont, MA (US); and Jenna Carroll Soper, Winchester, MA (US)
Assigned to Voyager Therapeutics, Inc., Lexington, MA (US)
Filed by Voyager Therapeutics, Inc., Cambridge, MA (US)
Filed on Mar. 22, 2022, as Appl. No. 17/701,238.
Application 17/701,238 is a continuation of application No. 16/756,595, abandoned, previously published as PCT/US2018/056001, filed on Oct. 16, 2018.
Claims priority of provisional application 62/666,934, filed on May 4, 2018.
Claims priority of provisional application 62/578,735, filed on Oct. 30, 2017.
Claims priority of provisional application 62/572,702, filed on Oct. 16, 2017.
Prior Publication US 2022/0211744 A1, Jul. 7, 2022
Int. Cl. C07H 21/04 (2006.01); A61K 9/00 (2006.01); A61K 31/713 (2006.01); C07H 21/02 (2006.01); C12N 15/113 (2010.01); C12N 15/63 (2006.01); C12N 15/85 (2006.01); C12N 15/86 (2006.01); A61P 25/28 (2006.01)
CPC A61K 31/713 (2013.01) [A61K 9/0085 (2013.01); C12N 15/113 (2013.01); C12N 15/63 (2013.01); C12N 15/85 (2013.01); C12N 15/86 (2013.01); A61P 25/28 (2018.01); C12N 2310/11 (2013.01); C12N 2310/14 (2013.01); C12N 2320/30 (2013.01)] 24 Claims
 
1. A method for inhibiting the expression of the superoxide dismutase 1 (SOD1) gene in a cell in a subject comprising administering to the subject an effective amount of an adeno-associated viral (AAV) particle comprising a viral genome and a capsid, wherein said viral genome comprises a modulatory polynucleotide sequence positioned between two inverted terminal repeats (ITRs), wherein said modulatory polynucleotide sequence comprises a nucleic acid sequence encoding a sense strand sequence and an antisense strand sequence of a siRNA duplex, and wherein the sense strand sequence comprises at least 20 consecutive nucleotides from the nucleotide sequence of SEQ ID NO: 2, and the antisense strand sequence comprises at least 20 consecutive nucleotides from the nucleotide sequence of SEQ ID NO: 1, thereby inhibiting expression of the SOD1 gene in the cell in the subject.