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US 12,305,191 B2Granted
Compositions and methods for treating non-age-associated hearing impairment in a human subject
AssigneeAkouos, Inc.
InventorsEmmanuel John Simons, Robert Ng
Life of this patent
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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.
Claims26
A plurality of recombinant adeno-associated (rAAV) vectors comprising:
- a) a first rAAV vector comprising, in order of 5′ to 3′:
- (i) a 5′ ITR comprising the sequence of SEQ ID NO:
- 97;
- (ii) a CAG promoter comprising the sequence of SEQ ID NO:
- 61;
- (iii) a 5′ OTOF coding region comprising exons 1 to (and through) 21 of OTOF cDNA, wherein the 5′ OTOF coding region is at least 90% identical to SEQ ID NO:
- 101 and encodes the same amino acid sequence as encoded by SEQ ID NO:
- 101;
- (iv) a SD intron sequence;
- (v) an AK recombinogenic region comprising the sequence of SEQ ID NO:
- 103;
- and
- (vi) a 3′ ITR comprising the sequence of SEQ ID NO:
- 104;
- and
- b) a second rAAV vector comprising, in order of 5′ to 3′:
- (i) a 5′ ITR comprising the sequence of SEQ ID NO:
- 97;
- (ii) an AK recombinogenic region comprising the sequence of SEQ ID NO:
- 103;
- (iii) a SA intron sequence;
- (iv) a 3′ OTOF coding region that comprises exons 22 to (and through) exon 48 of OTOF cDNA, wherein the 3′ OTOF coding region is at least 90% identical to SEQ ID NO:
- 107 and encodes the same amino acid sequence as encoded by SEQ ID NO:
- 107;
- (v) a polyA sequence;
- and
- (vi) a 3′ ITR comprising the sequence of SEQ ID NO:
- 104.
25 more elements
A composition comprising a first rAAV vector encapsidated by an Anc80 capsid and a second rAAV vector encapsidated by an Anc80 capsid, wherein:
- a) the first rAAV vector comprises, in order of 5′ to 3′:
- (i) a 5′ ITR comprising the sequence of SEQ ID NO:
- 97,
- (ii) a CAG promoter comprising the sequence of SEQ ID NO:
- 61,
- (iii) a 5′ OTOF coding region that comprises exons 1 to (and through) 21 of OTOF cDNA, wherein the 5′ OTOF coding region is at least 95% identical to SEQ ID NO:
- 101 and encodes the same amino acid sequence as encoded by SEQ ID NO:
- 101,
- (iv) a SD intron sequence,
- (v) an AK recombinogenic region comprising the sequence of SEQ ID NO:
- 103, and
- (vi) a 3′ ITR comprising the sequence of SEQ ID NO:
- 104;
- and
- b) the second rAAV vector comprises, in order of 5′ to 3′:
- (i) a 5′ ITR comprising the sequence of SEQ ID NO:
- 97,
- (ii) an AK recombinogenic region comprising the sequence of SEQ ID NO:
- 103,
- (iii) a SA intron sequence,
- (iv) a 3′ OTOF coding region that comprises exons 22 to (and through) exon 48 of OTOF cDNA, wherein the 3′ OTOF coding region is at least 95% identical to SEQ ID NO:
- 107 and encodes the same amino acid sequence as encoded by SEQ ID NO:
- 107,
- (v) a polyA sequence, and
- (vi) a 3′ ITR comprising the sequence of SEQ ID NO:
- 104;
- wherein the composition further comprises one or more pharmaceutically acceptable carriers, diluents, or excipients.
24 more elements
Dependencies are inferred from the claim text.