- RNA-lipid nanoparticle (RNA-LNP) medicines are a proven manufacturable and scalable modality for genomic medicines, enabling silencing, expression, and editing specific genes
- Precision NanoSystems has developed a Genomic Medicine Toolbox, comprising of lipid reagents, as well as the NanoAssemblr® microfluidic manufacturing platform, enabling end-to-end development of RNA-LNPs
RNA can be designed to silence, express, and edit specific genes providing a flexible and powerful approach to treating or preventing diseases. The recent success of RNA-lipid nanoparticle (RNA-LNP) COVID-19 vaccines has exemplified the massive potential and need to rapidly develop and scale up new genomic medicines to protect from emerging viral threats and treat a wide range of serious diseases with unmet medical needs.
Due to the RNA degradation and low cellular uptake, an essential element of genomic medicines are LNP-based delivery systems. Precision NanoSystems has developed a Genomic Medicine Toolbox for the end-to-end development of RNA-LNPs. The toolbox comprises off-the-shelf and proprietary lipid nanoparticle reagents, the NanoAssemblr® microfluidic nanoparticle manufacturing platform, and in-house expertise in formulation and process development. In this presentation, we provide examples of how these platform technologies are enabling (bio)pharma companies and research institutions to rapidly discover new RNA-LNP vaccines, gene therapies, and cell therapies and seamlessly scale them towards the clinic.
Viet Anh Huu Nguyen, Senior Field Application Scientist, Precision NanoSystems Inc.