From Liposomes over Lipoplexes to LNPs and back: Nanoparticle Drug Delivery Technologies for RNA

3/22/2022 09:20 - 09:50

The successful development of messenger RNA (mRNA)-based COVID-19 vaccines in extraordinarily short time has attracted great public attention to both, messenger RNA (mRNA) as a therapeutic molecule, and nanoparticles for drug delivery. For both, this was made possible due to previous, long-time, research in these fields, with liposomes and DNA-lipoplexes as earlier systems in development. There are some common features, which are shared by most of these products: (i) they are complex colloidal systems by nature, (ii) they are obtained by self-assembly processes (e.g. between RNA and positively charged lipids), (iii) the properties may sensitively depend on molecular composition, characteristics of the bulk phase and process parameters.

Insight obtained from basic research on biomembranes and concepts from colloid and interface sciences can be helpful for a better understanding of these drug delivery systems and thus provide a contribution for rational pharmaceutical development. Extended physicochemical characterization, including advanced techniques such as SAXS/SANS measurements, allow to reveal structural and functional coherencies inside these systems, which can be relevant to specify product characteristics and quality-related parameters. In this presentation, taking selected nanoparticle formats for RNA delivery as examples, key characteristics are described, and options to correlate physicochemical parameters to activity are outlined.

Dr. Heinrich Haas, Vice President Formulation & Drug Delivery , BioNTech