Route: Personalised medicine: the individual at the centre

Cluster question: 098 How can we use breakthroughs in basic biomedical research to develop new medicines?

Nucleic acid therapeutics are revolutionizing medicine. However, manufacture, scale-up, distribution and stability are the new limiting factors. NANOSPRESSO-NL aims to lay the groundwork for personalized and local production in a hospital pharmacy of nucleic acid therapeutics for orphan diseases. Most of the current state-of-the-art nucleic acid therapeutics, for example the clinically used siRNA-based drug formulation patisiran, are prepared by careful and controlled mixing of excipients and nucleic acid that electrostatically and hydrophobically complex into the final nanomedicine formulation. Since all therapeutic nucleic acids are essentially the same molecule -only with different lengths and sequences- the production process to turn them into nanomedicines is uniquely qualified for platform design. As a result, quality control can be the production process rather than the end product, similar to a popular method of local high-quality espresso making. This would allow rapid affordable local development of personalized therapeutics without distribution and stability issues.


drug regulatory science, mRNA, Nanomedicine, nucleic acid therapeutics, orphan diseases, patent law, Personalized medicine, pharmaceutical technology, siRNA


Organisation University Medical Center Utrecht (UMC Utrecht)
Name Prof. dr. R.M. (Raymond) Schiffelers