UPGRADE brings together the required expertise from different areas to generate a set of disruptive new technologies and transforming solutions to the major hurdles that have long constrained wider application of gene therapy.
Expected output comprise new targeted genome/epigenome editing strategies with high efficiency and precision, and new viral and non-viral vehicles for immune stealth targeted delivery to specific cells and tissue types.
All together these advances will generate novel AMPs to be tested and validated in relevant disease models. These prototype AMPs and the preclinical data generated by UPGRADE will create a solid foundation for launching new first-in-human trials after completion of the project.