Lipid nanoparticle (LNP) mRNA drug delivery systems
ARCT-810 Clin2 ornithine transcarbamylase deficiency; LUNAR -GSDIII Clin1/Clin2 glycogen storage disease type III (partner: Ultragenyx); LUNAR-CF Clin1 cystic fibrosis; LUNAR-FLU Clin0 influenza
Arcturus Therapeutics is using RNA technology to develop rare and infectious diseases therapeutics. Different forms of RNA can be used as a protein replacement therapy to treat diseases that lack a protein, gene editing, gene silencing and as vaccines. Arcturus is capitalizing on these strategies to create RNA medicines. The main challenges are identifying the most potent and specific RNA sequence and delivery of the therapeutic RNA to the desired cells. To achieve this, Arcturus is employing their Lipid-Mediated Delivery Enables Multiple RNA Medicines (LUNAR®) technology, a proprietary multi-component drug delivery system that is versatile, scalable and reproducible. Arcturus has leveraged their LUNAR platform to create a vaccine for COVID-19 which is in Phase 3 clinical trials.