US 12,491,223 B2
Method for treating an auditory neuropathy spectrum disorder
Yen-Fu Cheng, Taipei (TW); Chen-Chi Wu, Taipei (TW); Ying-Chang Lu, Taipei (TW); Robert Ng, Newton, MA (US); and Danielle R. Lenz, Brookline, MA (US)
Assigned to Akouos, Inc., Indianapolis, IN (US); Taipei Veterans General Hospital, Taipei (TW); and National Taiwan University Hospital, Taipei (TW)
Appl. No. 17/280,786
Filed by AKOUOS, INC., Indianapolis, IN (US); Taipei Veterans General Hospital, Taipei (TW); and National Taiwan University Hospital, Taipei (TW)
PCT Filed Sep. 27, 2019, PCT No. PCT/US2019/053465
§ 371(c)(1), (2) Date Mar. 26, 2021,
PCT Pub. No. WO2020/069320, PCT Pub. Date Apr. 2, 2020.
Claims priority of provisional application 62/737,406, filed on Sep. 27, 2018.
Prior Publication US 2022/0000972 A1, Jan. 6, 2022
Int. Cl. A61K 35/761 (2015.01); A61K 35/76 (2015.01); A61K 38/17 (2006.01); A61K 48/00 (2006.01); A61P 27/16 (2006.01); C07K 14/47 (2006.01); C12N 15/85 (2006.01)
CPC A61K 35/761 (2013.01) [A61K 35/76 (2013.01); A61K 38/1703 (2013.01); A61K 48/0058 (2013.01); A61K 48/0075 (2013.01); A61K 48/0083 (2013.01); A61P 27/16 (2018.01); C07K 14/47 (2013.01); C12N 15/8509 (2013.01); A61K 48/00 (2013.01)] 10 Claims
 
1. A method for treating an auditory neuropathy spectrum disorder (ANSD) in a subject comprising:
administering to the subject an adeno-associated virus (AAV) vector comprising: (i) an Anc80 capsid protein; and (ii) a DFNB59 gene,
wherein the subject has a mutant DFNB59 gene that results in a G292R mutation in a Pejvakin (PJVK) protein, and wherein the mutant DFNB59 gene comprises the nucleotide sequence of SEQ ID NO: 2.