US 11,807,867 B2
Compositions and methods for treating non-age-associated hearing impairment in a human subject
Emmanuel John Simons, Boston, MA (US); and Robert Ng, Boston, MA (US)
Assigned to Akouos, Inc., Boston, MA (US)
Filed by Akouos, Inc., Boston, MA (US)
Filed on Jul. 29, 2022, as Appl. No. 17/816,305.
Application 17/816,305 is a continuation of application No. PCT/US2021/018919, filed on Feb. 19, 2021.
Claims priority of provisional application 62/979,792, filed on Feb. 21, 2020.
Prior Publication US 2023/0108913 A1, Apr. 6, 2023
Int. Cl. C12N 15/86 (2006.01); A61P 27/16 (2006.01); C07K 14/47 (2006.01); A61K 48/00 (2006.01)
CPC C12N 15/86 (2013.01) [A61P 27/16 (2018.01); C07K 14/47 (2013.01); A61K 48/00 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14144 (2013.01)] 28 Claims
 
1. A plurality of recombinant adeno-associated viral (rAAV) vectors comprising:
a) a first rAAV vector comprising a nucleic acid sequence of SEQ ID NO: 96; and
b) a second rAAV vector comprising a nucleic acid sequence of SEQ ID NO: 105.
 
10. A method of expressing a recombinant full-length otoferlin protein in a mammalian cell, the method comprising administering a plurality of recombinant adeno-associated viral (rAAV) vectors comprising:
a) a first rAAV vector comprising a nucleic acid sequence of SEQ ID NO: 96; and
b) a second rAAV vector comprising a nucleic acid sequence of SEQ ID NO: 105;
to the mammalian cell, wherein the mammalian cell has reduced expression, lack of expression or dysfunction of otoferlin.
 
19. A method of treating hearing loss in a subject having otoferlin related hearing loss, the method comprising administering a plurality of recombinant adeno-associated viral (rAAV) vectors comprising:
a) a first rAAV vector comprising a nucleic acid sequence of SEQ ID NO: 96; and
b) a second rAAV vector comprising a nucleic acid sequence of SEQ ID NO: 105;
into the cochlea of the subject, wherein the subject has reduced expression, lack of expression, or dysfunction of otoferlin.