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US 2026/0201393 A1Published application
Compositions and methods for treating non-age-associated hearing impairment in a human subject
AssigneeAkouos, Inc.
InventorsEmmanuel John SIMONS, Ellen REISINGER, Sebastian KÜGLER, Hanan AL-MOYED
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Abstract
Provided herein are compositions that include at least two different nucleic acid vectors, where each of the at least two different vectors includes a coding sequence that encodes a different portion of an otoferlin protein, and the use of these compositions to treat hearing loss in a subject.
Claims30
1-63. (canceled)
A method of treating otoferlin related hearing loss, comprising administering to
- an inner hair cell a plurality of AAV vectors comprising:
- a first AAV vector comprising a first 5′ inverted terminal repeat (ITR), a promoter operably linked to a portion of a gene encoding a N-terminal portion of a human otoferlin isoform 5 polypeptide, a first splicing sequence, a first region of homology, and a first 3′ ITR;
- and
- a second AAV vector comprising a second 5′ ITR, a second region of homology, a second splicing sequence, a portion of the gene encoding a C-terminal portion of the human otoferlin isoform 5 polypeptide, a polyadenylation signal, and a second 3′ ITR;
- wherein the first region of homology is homologous to the second region of homology;
- wherein the first AAV vector and the second AAV vector are capable of constituting a full-length human otoferlin isoform-5 messenger RNA in the inner hair cell;
- and
- wherein the otoferlin related hearing loss is associated with reduced expression, lack of expression, or dysfunction of otoferlin.
5 more elements
A plurality of AAV vectors comprising:
- a first AAV vector comprising a first 5′ inverted terminal repeat (ITR), a promoter operably linked to a portion of a gene encoding a N-terminal portion of a human otoferlin isoform 5 polypeptide, a first splicing sequence, a first region of homology, and a first 3′ ITR;
- and
- a second AAV vector comprising a second 5′ ITR, a second region of homology, a second splicing sequence, a portion of the gene encoding a C-terminal portion of the human otoferlin isoform 5 polypeptide, a polyadenylation signal, and a second 3′ ITR;
- wherein the first region of homology is homologous to the second region of homology;
- and
- wherein the first AAV vector and the second AAV vector are capable of constituting a full-length human otoferlin isoform-5 messenger RNA in an inner hair cell.
3 more elements
A pharmaceutical composition comprising
- (a) a plurality of AAV vectors comprising:
- a first AAV vector comprising a first 5′ inverted terminal repeat (ITR), a promoter operably linked to a portion of a gene encoding a N-terminal portion of a human otoferlin isoform 5 polypeptide, a first splicing sequence, a first region of homology, and a first 3′ ITR;
- and
- a second AAV vector comprising a second 5′ITR, a second region of homology, a second splicing sequence, a portion of the gene encoding a C-terminal portion of the human otoferlin isoform 5 polypeptide, a polyadenylation signal, and a second 3′ ITR;
- wherein the first region of homology is homologous to the second region of homology;
- and
- wherein the first AAV vector and the second AAV vector are capable of constituting a full-length human otoferlin isoform-5 messenger RNA in the inner hair cell;
- and
- (b) one or more pharmaceutically or physiologically acceptable carriers, diluents or excipients.
6 more elements
Dependencies are inferred from the claim text.