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US 2026/0201344 A1Published application
Stable production systems for adeno-associated virus production
AssigneeAsimov Inc.
InventorsMichael T. Leonard, Jeremy J. Gam, Alec A.K. Nielsen
Life of this patent
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Abstract
Disclosed herein are genetically engineered cells for AAV production. The genetically engineered cell comprises molecular systems for temporal control of expression of genes required for AAV production. Also disclosed herein are methods of using genetically engineered cells for AAV production.
Claims64
An engineered cell for AAV production, comprising one or more stably integrated nucleic acid molecules collectively comprising a nucleic acid sequence encoding for each of:
- Rep52, DA-Rep52, Rep40, or DA-Rep40;
- Rep78, DA-Rep78, Rep68, or DA-Rep68;
- E2A or DA-E2A;
- E4ORF6 or DA-E4ORF6;
- VARNA or DA-VARNA;
- VP1 or DA-VP1;
- VP2 or DA-VP2;
- VP3 or DA-VP3;
- AAP or DA-AAP;
- MAAP or DA-MAAP;
- L4 100K or DA-L4 100K;
- and a Base Editor, each nucleic acid molecule being operably linked to a promoter;
- wherein the cell comprises the nucleic acid sequence of at least one of DA-Rep52, DA-Rep40, DA-Rep78, DA-Rep68, DA-E2A, DA-E4ORF6, DA-VP1, DA-VP2, DA-VP3, DA-AAP, DA-MAAP, and DA-L4 100K;
- wherein the nucleic acid sequences of DA-Rep52, DA-Rep40, DA-Rep78, DA-Rep68, DA-E2A, DA-E4ORF6, DA-VP1, DA-VP2, DA-VP3, DA-AAP, DA-MAAP, and DA-L4 100K each comprises a mutated start codon.
11 more elements
Dependencies are inferred from the claim text.