Design

EXPERT will design modular nanocarriers consisting of an mRNA payload, nanocarrier materials and, where applicable, a targeting ligand. The mRNA payload is a fragile molecule that bears the therapeutic activity. It can easily be destroyed by enzymes and has difficulties in reaching the site of activity. Therefore, the consortium will investigate the use of materials to protect and deliver the mRNA, including established, emerging and exploratory components which could be suitable for GMP* production.

• GMP or Good Manufacturing Practice is a system set out by relevant agencies, such as the European Medicines Agency, describing the minimum standard for processes, procedures and documentation that a medicines manufacturer must meet.

Production

The modular nanocarriers will be manufactured through quality-by-design with excellent control over the physicochemical parameters, reproducibility and scalability, which is compatible with GMP production. To remove operator variability, enable seamless scale-up and allow for robust manipulation of a single variable per experiment, EXPERT will have rapid mixing manufacturing at all partners’ research sites. The well-characterised, reproducible nanocarriers will subsequently be validated for safety and efficacy in in vitro and in vivo experiments.

Safety & Efficacy

To evaluate the performance, EXPERT will investigate the tissue distribution, cellular targeting and cytoplasmic translocation of promising modular nanocarriers. A pre-clinical in vitro and in vivo safety assessment as well as in vivo checks of the mRNA nanomedicine translation will help to analyse the therapeutic efficacy to select the best mRNA formulation for clinical evaluation.

Preparation for Clinical Study

EXPERT will prepare mRNA nanomedicine that is ready to be tested in a clinical trial in cancer patients.