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，开创了突破性的骨再生非病毒基因治疗。在前沿基础研究的支持下，TherageniX的肽质粒DNA复合物已被证明在体外、离体和临床前测试中以液体形式有效。这种液体形式可以支持更快的骨再生和减少恢复时间。TherageniX/UoN进行了广泛的概念验证研究，证明了通过喷雾和冷冻干燥制造干燥配方的潜在应用。在这个项目中，TherageniX将开发其专有的非病毒基因传递系统，将其实验性液体形式转化为增强的干燥药物配方，基因治疗粉末（GTP）。与英国政府推动细胞和基因治疗行业制造创新的政策密切相关，新配方将提高这种创新技术的效果效率（减少剂量以达到效果）并提高生产率（扩大生产规模以达到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.