California-based Arcturus Therapeutics announced that it will develop its ARCT-032 aerosolized CFTR mRNA formulation for the treatment of cystic fibrosis. According to Arcturus, preclinical studies of ARCT-032, which is based on the company’s LUNAR (lipid-enabled and unlocked nucleomonomer agent modified RNA) technology, have demonstrated that the inhaled therapy has the potential to successfully deliver CFTR mRNA to airway epithelial cells, which could result in the re-establishment of normal CFTR function in the lungs of CF patients.
Arcturus Chief Scientific Officer and Chief Operating Officer Pad Chivukula commented, “We are pleased to have advanced ARCT-032 as a novel mRNA-based development candidate for CF Lung Disease. ARCT-032, based on our proprietary LUNAR technology, is designed to result in the efficient expression of a functional cystic fibrosis transmembrane conductance regulator (CFTR) protein in the lungs. We believe that ARCT-032 has the potential to address the root cause of CF lung disease, which is caused by the defective, or missing, CFTR protein. While there has been progress with the recent approval of drugs for CF, many patients remain underserved. ARCT-032 has the potential to provide benefit to all CF patients, regardless of their underlying genetic mutations.”
Read the Arcturus Therapeutics press release.
