Spark Therapeutics, Inc.

Lin Lu

John Fraser Wright

Guang Qu

Younghoon Oh

Jesusa Josue-almqvist

Provided are compositions and methods of transducing/transfecting cells with a molecule, such as a nucleic acid (e.g., plasmid), at high efficiency. High efficiency transduced/transfected cells can, when transduced with a nucleic acid that encodes a protein or comprises a sequence that is transcribed into a transcript of interest, produce high amounts of protein and/or transcript. High efficiency transduced/transfected cells can, when transduced with plasmids comprising (i) nucleic acids encoding AAV packaging proteins and/or nucleic acids encoding helper proteins; and (ii) a transgene that encodes a protein or is transcribed into a transcript of interest; produce high amounts of recombinant rAAV vector.

Enhancing agents for improved cell transfection and/or rAAV vector production

In accordance with the invention, provided herein are methods for purifying recombinant adeno-associated (rAAV) vector particles.

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Wayne, PA, United States of America

Lin Lu