Business Development

ARCT-810 is an intravenously administered investigational mRNA therapeutic designed to express normal functional OTC enzyme in the liver of individuals with OTC deficiency. ARCT-810 has received Orphan Medicinal Product Designation and an approved pediatric investigation plan (PIP) from the European Medicines Agency (EMA), and Orphan Drug Designation, Fast Track Designation along with Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for the treatment of OTC deficiency. OTC is a key enzyme in the urea cycle which converts toxic ammonia into urea. Elevated ammonia can lead to metabolic crises with progressive and irreversible neurocognitive damage. A safe and effective mRNA therapeutic may restore normal functional OTC enzyme in the liver which could improve urea cycle activity, reduce abnormally elevated glutamine, maintain normal ammonia levels and potentially eliminate the risk of future metabolic crises. ARCT-810 is based on Arcturus’ mRNA design construct and proprietary manufacturing process. ARCT-810 also utilizes Arcturus’ extensive and propriety lipid library and employs the Company's LUNAR^® delivery platform to deliver OTC mRNA to hepatocytes.

ARCT-032 is an inhaled investigational mRNA therapeutic designed to express normal functional CFTR in the lungs of individuals with CF. ARCT-032 has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) and Orphan Drug Designation along with Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) to treat cystic fibrosis. ARCT-032 utilizes Arcturus’ LUNAR^® lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs. Lung disease is the leading cause of morbidity and mortality in people with CF. Expression of a functional copy of the CFTR mRNA in the lungs of people with CF has the potential to restore CFTR activity and mitigate the downstream effects that cause progressive lung disease. The ARCT-032 program is supported by preclinical data in rodents, ferrets and primates, as well as the demonstration of restoration of CFTR expression and function ex-vivo in human bronchial epithelial cells.