Robust Low-Volume Production For Screening High-Value Nanoparticle Materials

By Chelsea Cayabyab, Andrew Brown, Grace Tharmarajah, Anitha Thomas

In 2018, the FDA approval of Patisiran, a lipid nanoparticle (LNP) formulation and the first small interfering RNA therapeutic to receive FDA approval, established LNPs as the premier technology for non-viral RNA delivery. Concurrently, the NanoAssemblr^® platform, which harnesses microfluidic mixing, has been demonstrated as a simple, robust and scalable production method for LNPs encapsulating various types of nucleic acids with near 100% encapsulation efficiencies. LNPs provide a versatile option for gene knockdown or gene expression studies in vitro and in vivo. The NanoAssemblr Spark™ exploits an additional advantage of microfuidic mixing: microliter-scale formulation that conserves high-value materials such as novel lipids and mRNA. This unique combination of ultra-low volume formulations with a rapid, simple and reproducible process makes Spark an ideal platform for screening and early preclinical development of mRNA-LNP formulations. Effective screening programs can significantly narrow the parameter space for developing and optimizing next-generation delivery technologies and nanoparticle therapeutics.