Health; Destination 5: Unlocking the full potential of new tools, technologies and digital solutions for a healthy society
Next generation advanced therapies to treat highly prevalent and high burden diseases with unmet medical needs
Scope: This topic aims to ensure that the next wave of advanced therapies, based on either pluripotent stem cells, gene editing or RNA, are established in a timely fashion and in accordance with the appropriate regulatory standards for further clinical testing. It will support preclinical research platforms for disorders with high prevalence and burden that tackle the bottlenecks currently encountered in the field, ensuring that promising advanced therapies can reach the market within the next decade.
Method development for the production and differentiation of pluripotent stem cells (defined as cells that can give rise to cells from all three embryonic germ layers), to include defining appropriate potency assays. Complementary activities to assess mode of action, safety, in vivo validation or upscaling procedures could be considered.
Project budget: 6M (Total budget 60M); Deadline: 1 February 2022 (1st stage), 6 September 2022 (2nd stage). More