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

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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.