公开(公告)号：US20190292561A1

公开(公告)日：2019-09-26

申请号：US15780542

申请日：2016-12-01

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Guang QU ,  Lin LU ,  John Fraser WRIGHT

IPC: C12N15/86 ,  C12N5/00 ,  C12N5/073 ,  C12N5/071

Abstract: Methods and compositions for transfecting cells with plasmids are disclosed. In certain embodiments, methods and compositions are disclosed in which transfection efficiency is significantly increased by contacting the cells being transduced with polyethyleneimine (PEI) that is free of nucleic acid during the transfection process. Therapeutically useful adeno-associated viral vectors generated according to the disclosed methods and compositions are also disclosed.

公开(公告)号：US20200299650A1

公开(公告)日：2020-09-24

申请号：US16088743

申请日：2017-03-31

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Guang QU ,  Younghoon OH ,  Lin LU ,  John Fraser WRIGHT

IPC: C12N7/00 ,  C12N1/06 ,  B01D15/36 ,  B01D15/34 ,  B01D61/14 ,  B01D15/38

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

公开(公告)号：US20200165632A1

公开(公告)日：2020-05-28

申请号：US16619898

申请日：2018-06-06

Applicant: Spark Therapeutics, Inc.

Inventor： Guang QU ,  Lin LU ,  Jesusa JOSUE-ALMQVIST ,  John Fraser WRIGHT

IPC: C12N15/87 ,  C12N5/073

Abstract: 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.