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US 2025/0108130 A1Published application
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.
Claims32
A nucleic acid, comprising one or more helper polynucleotides, wherein the one or more helper polynucleotides comprise adeno-associated virus (AAV) Rep comprising the nucleic acid sequence of SEQ ID NO:
- 10 or AAV Cap comprising the nucleic acid sequence of SEQ ID NO:
- 11, and wherein the one or more helper polynucleotides comprise reduced CpG dinucleotides as compared to a parental equivalent.
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A recombinant AAV vector comprising:
- a) a first nucleic acid comprising a nucleotide sequence having at least 90% sequence identity to the nucleic acid sequence of SEQ ID NOs:
- 13 or 14;
- and
- b) a second nucleic acid comprising one or more helper polynucleotides, the first helper polynucleotide of the one or more helper polynucleotides comprising AAV Rep comprising the nucleic acid sequence of SEQ ID NO:
- 10;
- and the second helper polynucleotide of the one or more helper polynucleotides comprising AAV Cap comprising the nucleic acid sequence of SEQ ID NO:
- 11.
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A pharmaceutical composition comprising:
- a) a recombinant AAV vector comprising a payload of interest;
- b) a plurality of helper polynucleotides, comprising adeno-associated virus (AAV) Rep comprising the nucleic acid sequence of SEQ ID NO:
- 10 and/or AAV Cap comprising 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.
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Dependencies are inferred from the claim text.