PFI:AIR - TT: Prototyping a Gene Transfection Tool, GeneTrain

New York University, New York NY

Investigators

Abstract

This PFI: Accelerating Innovation Research (AIR) Technology Translation (TT) project focuses on translating an engineered protein and lipid mixture that addresses the need for highly efficient delivery of nucleic acids such as siRNA and DNA into living cells. Delivery of nucleic acids into cells can be used as a method to treat certain diseases or study natural cell behavior. This innovation, entitled GeneTrain, is important because existing chemicals that facilitate the delivery of nucleic acids into cells, known as transfection reagents, are effective with only certain types of cells. The technology proposed here will enable researchers to perform nucleic acid delivery experiments across many different types of cells thereby expediting and expanding the discovery of new siRNA or DNA-based therapies. In addition, such a robust research tool will allow scientists to accelerate biomedical research en route to a deeper understanding of diseases. This product has the following unique features: the novel design and application of an engineered protein bearing a highly positive charge for binding to negatively charged nucleic acid, resulting in efficient delivery of siRNA and plasmid DNA, as well as uniform effectiveness across a broad range of cell types. These features provide the end user and customer with a solution that fulfills a wide spectrum of experimental requirements, removing the necessity to use multiple reagents or spend countless hours optimizing cell culture conditions when compared to conventional methods. The project will result in a prototype of GeneTrain and addresses technology gaps of formulation optimization and cell line/competitor benchmarking, which both vastly hinder the commercialization progress of this innovation. The initial data, while promising, suggests that the level limits of formulation factors (engineered protein-lipid compositional ratios) have not yet been adequately probed so as to yield optimal effects on transfection efficiency. In addition to the multidisciplinary research environment, graduate students and post-doctoral fellows will receive entrepreneurship and technology translation experiences through direct and frequent communication with entrepreneurial mentors in the life sciences, university technology transfer liaisons, patent attorneys, and potential licensing partners/customers. The project engages the management staff of the NYU Entrepreneurial Institute, the NYU Office of Industrial Liaison, and Ascension Bio, LLC to assist in the professional development of personnel, to guide partnership formation and licensing negotiation, and to advise on customer and product development strategy, respectively, in this technology translation effort from research discovery toward commercial reality. Research partners at the inception of this innovation include the NYU Langone Medical Center and the NYU College of Dentistry, both providing in vitro and in vivo laboratory resources for assistive development of the commercial innovation.