Heinrich Haas, representing NeoVac and collaborating with Johannes Gutenberg University in Mainz, delivered a presentation on nanoparticle technologies for mRNA delivery. Haas began by providing an overview of the current state of lipid nanoparticles (LNPs), which are assembled from specific lipids and used in vaccines like those for COVID-19. He highlighted NeoVac's innovative approach, which expanded the molecular architecture of ionisable lipids by introducing cleavable, structural, and functional elements. This innovation allows for the creation of nanoparticles tailored for specific therapeutic interventions and organ targeting.
Haas emphasised the ability to tailor the immunogenicity of lipids, addressing the unmet need for lipids with favourable immunogenicity for various applications. This capability enables the selection of lipids with high or low immunogenicity, depending on the intended use, such as vaccination or therapeutic intervention. He also mentioned the completion of the first clinical trial with a nanoparticle formulation as a COVID-19 vaccine, noting that the technology demonstrated a superior safety profile compared to licensed products.
The presentation then shifted to the quality control of pharmaceutical nanoparticles, particularly mRNA nanoparticles. Haas explained that classical control strategies were insufficient for these colloidal products and stressed the need to develop analytical protocols that consider parameters like particle size distribution, free and bound RNA, and internal structures. He discussed the use of X-ray scattering as a key method for obtaining structural information at different length scales and its application in understanding the quality attributes of nanoparticles.
Haas also described the development of ultra-small saRNA nanoparticles as improved vaccines. He detailed the process of polyplex formation between RNA and polyethylene amine, which resulted in highly compact globular structures with increased in vitro activity. This innovation offered advantages for various therapeutic applications, including targeting compartments with small fenestration.
In conclusion, Haas underscored the importance of combining controlled self-assembly strategies with characterisation techniques to enhance the quality and efficacy of mRNA pharmaceuticals. He expressed optimism that these advancements would accelerate the development and practical use of next-generation mRNA-based therapies.
