Ribonucleic acid (RNA) based genetic medicines are a promising way to treat many diseases as these medicines can be highly personalized. However, there remain challenges to their development and widespread use. These include: 

• Getting RNA molecules into specific target cells in the body efficiently to reduce side effects 

• Improving their stability to produce a sustained therapeutic effect and  

• The methods available to assess these genetic medicines are unable to predict their effectiveness. Therefore, testing potential RNA therapeutics is usually labour intensive and cost-intensive, which slows drug optimization efforts.  

Led by Drs. Eric Jan and Sabrina Leslie, this research aims to address these challenges and advance the development of new genetic medicines for cystic fibrosis and autoimmune disorders. These genetic medicines will consist of therapeutic RNAs encapsulated in lipid nanoparticles (LNPs). This study proposes utilizing RNA elements identified from viruses to enhance the stability of RNA in human target cells, employing advanced imaging based techniques to evaluate the designed genetic medicines and using this data to develop mathematical models that may predict the effectiveness of RNA-based medicines.  

This research has the potential to accelerate the development of novel genetic therapies for diverse diseases, such as autoimmune disorders, cystic fibrosis, cancers and inherited disorders.