What’s next in RNA therapeutics: Process for Stability?
EducationalCHALLENGES IN CRYOPRESERVATION FOR CELL & GENE THERAPIES
2026-05-27 | 11:00 AM - 11:15 AM | TPD Room
Abstract
In the recent years, the field of RNA and DNA therapeutics has evolved as a ground-breaking approach in medical research. General challenges are related to the stability and delivery of oligonucleotides for use in drug products.
Nanotechnology is therefore playing a key role in addressing these needs for stabilization and enabling of RNA delivery for different applications, from vaccination to genetic diseases’ treatment. It is easy to think about the huge impact that lipid nanoparticles had during the Covid-19 pandemic, opening the door to a totally new vaccination concept that is currently being applied to flu and cancer, as recent research shows. Lipid and polymeric nanoparticles have clearly shown the advantages of stabilizing oligonucleotides for a more efficient, tissue-specific delivery. In addition, the use of nanotechnologies has certainly enabled the development of freeze and spray dried products with a longer shelf-life and no need for cold-chain.
However, when it comes to nanoparticles manufacturing more flexible, efficient and scalable processes are necessary in order to ensure a more sustainable and effective production with less material loss, and consequently lower costs, and higher in vivo efficacy.
In this talk we will explore a novel nanoparticles manufacturing technology to enable an easier, faster and scalable development of oligonucleotides-based therapeutics, showing how these delivery systems are also fundamental in obtaining long-term stable nucleotide powder products by traditional lyophilization or spray drying, ensuring preservation of the expensive, precious cargo. Aseptic production of dry powders comprising RNA nanoparticles and filling of these stable dry powders in dual chamber syringes or different primary packaging containers will be described and discussed too.
Finally, alternatives routes for delivery of these new modalities through stable dry powders will be listed and examined.
