Policy Paper
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Challenges

EXPERT will address several major challenges that have limited mRNA therapeutics from displaying their full potential:

  • The ability to achieve functional target tissue delivery of mRNA by nanomedicine design
  • The capability to circumvent adverse immune responses towards mRNA nanomedicines
  • The scientific knowledge to achieve general applicability of an mRNA-based platform technology for various diseases and administration routes
  • The know-how to accomplish quality control and overcome regulatory hurdles

Project Objectives

  • Development of off-the-shelf targeted mRNA nanomedicines
  • Production of mRNA nanomedicines in a quality-by-design (QbD) approach
  • Good Manufacturing Practice scale-up to be ready for clinical translation
  • Investigation of mRNA nanomedicines for heart failure patients