Granted Patent
B2
US 12,188,041 · App. 17/290,082 · Granted Jan 7, 2025
Codon optimized otoferlin AAV dual vector gene therapy
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Abstract
Provided herein are methods and compositions for expressing Otoferlin, e.g., utilizing adeno-associated viral (AAV) particles. Further provided herein are compositions of AAV particles comprising one or more polynucleotides encoding Otoferlin are codon optimized for expression in human cells. Such methods and compositions may be useful for treatment of diseases and disorders such as Deafness, Autosomal Recessive 9 (DFNB9). Also provided are kits comprising such compositions.
Claims (7)
1. A recombinant AAV nucleic acid vector comprising a nucleotide sequence having at least 90% identity to any one of the sequences set forth in SEQ ID NOs: 17-21.
2. A composition comprising an rAAV particle comprising one or more of the recombinant AAV nucleic acid vector of claim 1 .
3. The composition of claim 2 , wherein the composition comprises a first recombinant AAV nucleic acid vector comprising a nucleotide sequence having at least 90% identity to the sequence set forth in SEQ ID NO: 17 or 18, and a second recombinant AAV nucleic acid vector comprising a nucleotide sequence having at least 90% identity to the sequence set forth in SEQ ID NO: 19.
4. The composition of claim 2 , wherein the composition comprises a first recombinant AAV nucleic acid vector comprising a nucleotide sequence having at least 90% identity to the sequence set forth in SEQ ID NO: 20, and a second recombinant AAV nucleic acid vector comprising a nucleotide sequence having at least 90% identity to the sequence set forth in SEQ ID NO: 21.
5. A recombinant AAV nucleic acid vector comprising a nucleotide sequence that comprises any one of the sequences set forth in SEQ ID NOs: 17-21.
6. A recombinant AAV (rAAV) nucleic acid vector comprising a nucleotide sequence having at least 90% identity to the codon optimized portion of any one of the sequences set forth in SEQ ID NOs: 17-19, wherein the codon optimized portion comprises nucleotide positions 565-4152 of SEQ ID NO: 17 or SEQ ID NO: 18, or nucleotide positions 1153-3558 of SEQ ID NO: 19.
7. The rAAV nucleic acid vector of claim 6 comprising the nucleotide sequence of the codon optimized portion of any one of the sequences set forth in SEQ ID NOs: 17-19, wherein the codon optimized portion comprises nucleotide positions 565-4152 of SEQ ID NO: 17 or SEQ ID NO: 18, or nucleotide positions 1153-3558 of SEQ ID NO: 19.
Assignments (2)
CONFIRMATORY LICENSE
Recorded Dec 5, 2023
From: UNIVERSITY OF FLORIDA
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 065774/0277 →
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Aug 16, 2021
From: DYKA, FRANK M.; HAUSWIRTH, WILLIAM W.
To: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED
Reel/Frame 057186/0067 →
Continuity (2)
Provisional Application
62754458
· Nov 1, 2018
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