UPGRADE fully embraces this challenge and plans to develop radical new solutions to the hurdles that currently limit broad application of gene therapy. To this end, the consortium will develop disruptive innovative technologies for precision gene and epigenome editing and combine them with the most advanced delivery tools in order to generate novel prototypes of Advanced Medicinal Products (AMPs).
The safety and efficacy profile of each novel AMPs will be stringently validated in one or more disease models paradigmatic for urgent unmet medical need and potential long-term cure, if the limitations of current gene therapies are overcome.
We envision that each new AMP generated by UPGRADE represents a versatile product that could be adapted to the treatment of several diseases because of related pathogenetic mechanisms or correction strategies, thus providing the basis for tackling diseases and conditions affecting large patient groups.