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US 12,514,933 B2Granted
Modified CPG dinucleotides for recombinant viral vector production
AssigneeSiren Biotechnology, Inc.
InventorsNicole Kristen Paulk, Nathalie Andree Nancy Clement, Siti Nur Sarah Morris
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Abstract
The disclosure relates generally to methods of producing recombinant viral vectors with reduced immunogenicity.
Claims20
A nucleic acid, comprising two or more helper polynucleotides, wherein the two or more helper polynucleotides comprise an adeno-associated virus (AAV) Rep gene comprising the nucleic acid sequence of SEQ ID NO:
- 10 and an AAV Cap gene comprising the nucleic acid sequence of SEQ ID NO:
- 11, and wherein the two or more helper polynucleotides comprise reduced CpG dinucleotides as compared to a wild-type gene equivalent.
A recombinant adeno-associated virus (AAV) vector comprising:
- (a) a first nucleic acid comprising a payload of interest comprising a sequence 100% identical to the nucleic acid sequence of SEQ ID NO:
- 14;
- and
- (b) a second nucleic acid comprising two or more helper polynucleotides, a first helper polynucleotide of the two or more helper polynucleotides comprising an AAV Rep gene comprising a sequence 100% identical to the nucleic acid sequence of SEQ ID NO:
- 10;
- and a second helper polynucleotide of the two or more helper polynucleotides comprising an AAV Cap gene comprising a sequence 100% identical to the nucleic acid sequence of SEQ ID NO:
- 11.
4 more elements
A pharmaceutical composition comprising:
- (a) a recombinant adeno-associated virus (AAV) vector comprising a payload of interest;
- (b) a plurality of helper polynucleotides comprising an AAV Rep gene comprising a sequence 100% identical to the nucleic acid sequence of SEQ ID NO:
- 10 and an AAV Cap gene comprising a sequence 100% identical to the nucleic acid sequence of SEQ ID NO:
- 11, wherein the plurality of helper polynucleotides comprise reduced CpG dinucleotides;
- and
- (c) a pharmaceutically acceptable excipient.
3 more elements
Dependencies are inferred from the claim text.