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

该PFI：加速创新研究（AIR）技术转化（TT）项目的重点是翻译一种工程蛋白和脂质混合物，以满足将siRNA和DNA等核酸高效递送到活细胞中的需求。将核酸递送到细胞中可用作治疗某些疾病或研究天然细胞行为的方法。 这项名为GeneTrain的创新很重要，因为现有的促进核酸进入细胞的化学物质（称为转染试剂）只对某些类型的细胞有效。 本文提出的技术将使研究人员能够在许多不同类型的细胞中进行核酸递送实验，从而加快和扩大新的siRNA或基于DNA的疗法的发现。 此外，这种强大的研究工具将使科学家能够加速生物医学研究，从而更深入地了解疾病。 该产品具有以下独特功能：设计和应用了一种带有高度正电荷的工程蛋白，用于结合带负电荷的核酸，从而有效递送siRNA和质粒DNA，并在广泛的细胞类型中具有一致的效力。 这些功能为最终用户和客户提供了一种解决方案，可满足广泛的实验要求，与传统方法相比，无需使用多种试剂或花费无数小时优化细胞培养条件。该项目将产生GeneTrain原型，并解决配方优化和细胞系/竞争对手基准测试的技术差距，这两者都极大地阻碍了这项创新的商业化进程。 最初的数据，而有希望的，表明配方因素（工程蛋白质-脂质组成比）的水平限制尚未得到充分的探讨，以产生最佳的转染效率的影响。除了多学科的研究环境，研究生和博士后研究员将通过与生命科学领域的创业导师、大学技术转让联络员、专利律师和潜在的许可合作伙伴/客户进行直接和频繁的交流，获得创业和技术翻译经验。该项目吸引了纽约大学创业学院、纽约大学工业联络办公室、和Ascension Bio，LLC协助人员的专业发展，指导合作伙伴关系的形成和许可谈判，并分别就客户和产品开发战略提出建议，在这项技术从研究发现转化为商业现实的努力中。这项创新的研究合作伙伴包括纽约大学Langone医学中心和纽约大学牙科学院，两者都为商业创新的辅助开发提供体外和体内实验室资源。

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.