Gene therapy in a powder: World’s first powdered gene therapy for bone graft augmentation

TherageniX是一家总部位于英国的中小企业，与诺丁汉大学(UON)合作，开创了开创性的非病毒骨再生基因疗法。在尖端基础研究的支持下，TherageniX的多肽-质粒DNA复合体在体外、体外和临床前测试中以液体形式被证明是有效的。这种液体形式可以支持更快的骨骼再生，并减少恢复时间。TherageniX/UON已经进行了广泛的概念验证研究，展示了通过喷雾和冷冻干燥创造干性制剂的潜在应用。在该项目中，TherageniX将开发其专有的非病毒基因传递系统，将其实验液体形成转化为增强型干药制剂--粉末基因疗法(GTP)。与英国政府推动细胞和基因疗法行业制造创新的政策严格一致，新配方将提高效果效率(减少剂量以达到效果)，并提高这项创新技术的生产率(扩大生产到GMP)。

TherageniX is a UK-based SME pioneering ground-breaking non-viral gene therapy for bone regeneration in collaboration with the University of Nottingham (UoN).Underpinned by cutting-edge fundamental research, TherageniX's peptide-plasmid DNA complexes have been proven effective in liquid form in in vitro, ex vivo and pre-clinical testing. This liquid form can support faster bone-regeneration and reduce recovery times. TherageniX/UoN have conducted extensive proof-of-concept studies that have demonstrated the potential application of creating a dry formulation via spray- and freeze-drying.Within this project, TherageniX will develop its proprietary non-viral gene delivery system, transforming its experimental liquid formation into an enhanced dry drug formulation, Gene-therapy-in-a-Powder (GTP).Strongly aligned with the UK Government's policy to drive manufacturing innovation in the cell and gene therapy industry, the new formulation will improve effect efficiency (reduced dose to reach effect) and increase productivity (scale-up manufacturing to GMP) for this innovative technology.