Arcturus Therapeutics Holdings Inc. announced that the Clinical Trial Application (CTA) for ARCT-032, an inhaled investigational mRNA medicine to treat cystic fibrosis (CF), received approval to proceed into a Phase 1 First-in-Human study in New Zealand. Cystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the Cystic Fibrosis Transmembrane (CFTR) gene result in a reduction or absence of CFTR quantity and/or function in the airways, causing insufficient chloride transport to maintain airway surface homeostasis. CF mucus is more difficult to clear, thus clogging the airways and leading to infection, inflammation, respiratory failure, or other life-threatening complications. Currently approved CFTR modulator therapies are designed to increase function of the CFTR channel to help reduce symptoms yet are ineffective in many people with CF as a result of their underlying mutations.
ARCT-032 will utilize Arcturus' LUNAR® lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs. 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 demonstrating restoration of expression and function in human bronchial epithelial cells.