Polynucleotides for use in AAV production
AssigneeEncoded Therapeutics, Inc.
InventorsAnne Tanenhaus, Tulasi Solanki, Adam Miller, Ulrike Jung
Life of this patent
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Abstract
The disclosure provides adeno-associated virus (AAV) helper plasmid variants with improved safety profiles. In certain aspects, an AAV helper plasmid variant of the disclosure comprises one or more inactivating mutations in at least one of the following adenovirus genes or genome sequences: (a) adenoviral fiber gene; (b) adenoviral precursor terminal protein gene; (c) adenoviral L1-52K gene; (d) adenoviral 100K gene; (e) adenoviral PVIII gene; (f) adenoviral E4 region open reding frame; (g) adenoviral inverted terminal repeat (ITR) sequence; (h) L3-23K region gene; (i) hexon-assembly gene; or (j) a combination of any of (a)-(i).
Claims23
A polynucleotide comprising one or more adenoviral genes or genome regions, wherein one or more of the adenoviral genes or genome regions comprises one or more inactivating mutations, and wherein the one or more adenoviral genes or genome regions is selected from the group consisting of:
- (a) an adenoviral fiber gene;
- (b) an adenoviral precursor terminal protein (pTP) gene;
- (c) an adenoviral L1-52K gene;
- (d) an adenoviral 100K gene;
- (e) an adenoviral PVIII gene;
- (f) an adenoviral E4 region open reading frame (orf);
- (g) an adenoviral inverted terminal repeat (ITR) sequence;
- (h) an L3-23K region gene;
- (i) a hexon-assembly gene;
- and
- (j) a combination of any of (a)-(i).
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Dependencies are inferred from the claim text.