Arcturus Therapeutics Receives Orphan Drug Designation from the U.S. FDA for ARCT-032, for the Treatment of Cystic Fibrosis
First cystic fibrosis patient in Phase 1b study successfully completed two administrations of ARCT-032
On track to share interim Phase 1b data in H1 2024
“Orphan Drug Designation is a very important regulatory milestone in our development plan for ARCT-032,” said
The first CF patient in our Phase 1b study successfully completed two administrations of ARCT-032. We remain on track to share interim Phase 1b data in H1 2024.
About Cystic Fibrosis
Cystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene result in a reduction or absence of CFTR protein 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 some people with CF because of their underlying mutations.
ARCT-032 utilizes 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 CFTR expression and function in human bronchial epithelial cells.
Founded in 2013 and based in
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