COMPOSITIONS AND METHODS FOR REDUCING REVERSE PACKAGING OF CAP AND REP SEQUENCES IN RECOMBINANT AAV

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United States of America Patent

APP PUB NO 20230235353A1
SERIAL NO

17911601

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Abstract

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The present disclosure provides compositions including recombinant nucleic acid constructs, vectors, and host cells, and methods of their use for reducing reverse packaging of cap and/or rep DMA sequences in the production of recombinant adeno-associated vims (rAAV). Also provided are pharmaceutical compositions comprising an rAAV produced from a composition or method of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical compositions may be useful in gene therapy for the prevention or treatment of a disease, condition, or disorder in a subject.

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Patent OwnerAddress
ULTRAGENYX PHARMACEUTICAL INCNOVATO CA

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Camplin, Brady Quincy, US 1 0
Craig, Stewart Cambridge, US 20 1655
Fuller, Matthew Scott Millis, US 6 0
Wadsworth, Samuel Monterey, US 26 195

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