Therapy Areas: Hereditary Disorders
Arcturus Therapeutics to Advance ARCT-032 as a Development Candidate for Cystic Fibrosis Lung Disease
30 December 2020 - - US-based clinical-stage messenger RNA medicines company Arcturus Therapeutics Holdings Inc. (NASDAQ: ARCT) has selected ARCT-032, an aerosolized LUNAR mRNA-based therapeutic candidate, as a development candidate for Cystic Fibrosis, a progressive hereditary disease, the company said.

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 CF patients has the potential to restore normal lung CFTR activity.

The ARCT-032 program is supported by preclinical data in CFTR deficient murine model, ferrets and NHPs.

LUNAR-CFTR can be efficiently delivered to epithelial cells in the airways and restore chloride channel activity in a CFTR KO mice model following intranasal administration of LUNAR-CFTR.

An mRNA-based replacement therapy for CF, if successfully developed, has the potential to deliver a fully functional copy of CFTR into the lungs, independent of the underlying CF genotype.

The expression of functional CFTR protein is expected to restore chloride channel efflux in the airways, reducing mucus accumulation, tissue scarring, and minimizing the progressive respiratory dysfunction observed in CF patients.

Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (NASDAQ: ARCT) is a clinical-stage mRNA medicines and vaccines company with enabling technologies: LUNAR lipid-mediated delivery, STARR mRNA Technology and mRNA drug substance along with drug product manufacturing expertise.

Arcturus' diverse pipeline of RNA therapeutic and vaccine candidates includes self-replicating mRNA vaccine programs for SARS-CoV-2 (COVID-19) and Influenza, and other programs to potentially treat Ornithine Transcarbamylase Deficiency, Cystic Fibrosis, and Cardiovascular Disease along with partnered programs including Glycogen Storage Disease Type 3, Hepatitis B Virus, and non-alcoholic steatohepatitis.

Arcturus' versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, replicon RNA, antisense RNA, microRNA, DNA, and gene editing therapeutics.


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