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US 2026/0286386 A1Published application
Compositions and methods for recombinant aav production
AssigneeREGENXBIO Inc.
InventorsPing LIU, Ayda MAYER, Chih-Li LIN, Thilo H. POHLE, Yusuf B. JOHARI, David C. JAMES
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Abstract
Provided herein are recombinant polynucleotides and plasmids suitable for use in the production of recombinant AAV particles. Also provided herein are methods for producing rAAV particles.
Claims15
An isolated recombinant polynucleotide comprising
- a) a first nucleotide sequence encoding an adenovirus E2A DNA binding protein (DBP) operably linked to a first promoter;
- b) a second nucleotide sequence encoding an adenovirus E4 polypeptide operably linked to a second promoter;
- c) a third nucleotide sequence encoding an adenovirus VA RNA I;
- and
- d) a fourth nucleotide sequence encoding a parvovirus p5 promoter, which is optionally an adeno associated virus (AAV) p5 promoter, and a fifth nucleotide sequence encoding an AAV rep gene and an AAV cap gene, wherein the parvovirus p5 promoter is operably linked to the AAV rep gene and controls the expression of the rep78 and rep68 gene products,
- optionally wherein the isolated recombinant polynucleotide does not comprise a nucleotide sequence encoding an adenovirus ITR sequence, L3 23K endoprotease, L5 pVI/fibre, and/or L4 pVIII/hexon-associated precursor.
3 more elements
Dependencies are inferred from the claim text.