Formulation Matters: RNA Nanoparticle Formulation, Process, And Characterization

By R.K. DeLong, M.S., Ph.D.; and Eli Kraus, Ph.D.

Ribonucleic acid (RNA) plays a critical role in regulating and controlling molecular processes within cells and tissues. Over the years, the significance of RNA, particularly messenger RNA (mRNA), in personalized medicine and drug development has expanded rapidly. The use of RNA as a therapeutic option dates back to 1978, when mRNA was first delivered to mouse cells using liposomes. However, the first clinical breakthrough occurred in 2017, when Sahin et al. pioneered an RNA-based poly-neo-epitope approach to mobilize the immune system for melanoma treatment.

The COVID-19 pandemic has further accelerated public awareness of mRNA and its applications, especially in vaccine development. RNA-based therapeutics are becoming increasingly important in personalized medicine, offering promising treatments for a range of diseases. As these therapies advance, the delivery systems for RNA-based drugs continue to evolve, focusing on improving drug loading, stability, and targeting capabilities.

This white paper explores the current state-of-the-art in RNA nanoparticle formulation, examining the technologies and strategies used to optimize RNA delivery for preclinical and clinical development. As the field grows, understanding how to formulate RNA nanoparticles effectively is essential for advancing therapeutic innovations.