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

这个PFI：加速创新研究（AIR）技术转化（TT）项目专注于翻译一种工程蛋白和脂质混合物，以解决将核酸（如siRNA和DNA）高效递送到活细胞中的需求。将核酸输送到细胞中可以作为治疗某些疾病或研究自然细胞行为的一种方法。这项名为GeneTrain的创新技术非常重要，因为现有的用于将核酸输送到细胞中的化学物质，即转染试剂，只对某些类型的细胞有效。这里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传递实验，从而加速和扩大新的siRNA或dna治疗方法的发现。此外，这样一个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的特点：一种具有高正电荷的工程蛋白的新颖设计和应用，可与带负电荷的核酸结合，从而有效地递送siRNA和质粒DNA，以及在广泛的细胞类型中具有统一的有效性。这些功能为最终用户和客户提供了满足广泛实验要求的解决方案，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的原型，并解决配方优化和细胞系/竞争对手基准测试方面的技术差距，这两者都极大地阻碍了这项创新的商业化进程。初步数据虽然很有希望，但表明尚未充分探索配方因子（工程蛋白-脂质组成比）的水平限制，从而对转染效率产生最佳影响。除了多学科的研究环境外，研究生和博士后还将通过与生命科学领域的创业导师、大学技术转让联系人、专利律师和潜在的许可合作伙伴/客户直接和频繁的交流，获得创业和技术翻译经验。该项目聘请了纽约大学创业研究所、纽约大学工业联络办公室和阿森松生物有限责任公司的管理人员，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，并在技术从研究发现到商业现实的转化过程中分别为客户和产品开发策略提供建议。这项创新开始时的研究合作伙伴包括纽约大学朗格尼医学中心和纽约大学牙科学院，它们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.