The Children's Hospital of Philadelphia

Bernd Hauck

Katherine A High

John Fraser Wright

Guang Qu

Federico Mingozzi

Olga Zelenaia

Methods for preparing highly purified AAV vector formulations are provided. The highly pure AAV formulations described herein are superior for clinical use.

Scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy

In accordance with the present invention, a method for increasing the yield of rLV vector particles comprising a trans gene encoding a therapeutic protein or fragment thereof is disclosed. In one approach, cells are transfected with plasmids encoding the necessary components for rLV production using a calcium chloride transfection mix at pH 7.1 wherein the calcium chloride and plasmids form a complex which is added to the cells at a constant speed. The cells are then incubated for a suitable time period wherein virus particle media is collected at least twice during the incubation period and stored in a cold storage unit, thereby reducing virus inactivation.

Large commercial scale lentiviral vector production system and vectors produced thereby

This application relates to the fields of gene therapy and molecular biology. In accordance with the present invention, an adeno-associated virus (AAV) vector comprising an altered capsid protein is provided. More specifically, this invention provides adeno-associated viral vectors comprising protein capsid variants which accelerate vector breakdown and clearance, thereby reducing undesirable immune responses.

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Hamilton, NJ, United States of America

Bernd Hauck