Samenvatting

The NANOSPRESSO project is a pioneering response to the complex challenge of
treating orphan diseases, which, despite affecting millions of people globally, have
only scant therapeutic options. This initiative represents a paradigm shift by
decentralizing the production of personalized nucleic acid nanomedicines.
Integrating advanced microfluidic technology with lipid nanoparticle engineering
platforms—validated by their efficacy in COVID-19 messenger (m)RNA vaccines—
the NANOSPRESSO model enables hospital pharmacists to seamlessly assemble
tailored therapeutic cartridges for gene/RNA therapy administration at the patient’s
bedside. This innovative model subverts the traditional constraints of high-cost,
intricate manufacturing and the instability of nucleic acid-based treatments, offering
a streamlined. localized, flexible, and patient-centric alternative. Inspired by the
traditional art of compounding in pharmacy, NANOSPRESSO strives to democratize
access to innovative treatments for rare diseases, challenging the conventional,
monolithic medical approach.