Arcturus Therapeutics to Advance ARCT-032, an Aerosolized LUNAR® mRNA-based Therapeutic, as a Development Candidate for Cystic Fibrosis Lung Disease

SAN DIEGO, CA, USA I December 30, 2020 I Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a leading clinical-stage messenger RNA medicines company focused on the development of infectious disease vaccines and significant opportunities within liver and respiratory rare diseases, today announced that it has selected ARCT-032, an aerosolized LUNAR® mRNA-based therapeutic candidate, as a development candidate for Cystic Fibrosis (CF), a progressive hereditary disease.

“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,” said Pad Chivukula, Ph.D. Chief Scientific Officer and Chief Operating Officer of Arcturus Therapeutics. “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.”

About ARCT-032

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.

About Arcturus Therapeutics

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: (i) LUNAR® lipid-mediated delivery, (ii) STARR™ mRNA Technology and (iii) 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 (OTC) Deficiency, Cystic Fibrosis, and Cardiovascular Disease along with partnered programs including Glycogen Storage Disease Type 3, Hepatitis B Virus, and non-alcoholic steatohepatitis (NASH). 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. Arcturus’ technologies are covered by its extensive patent portfolio (200 patents and patent applications, issued in the U.S., Europe, Japan, China and other countries). Arcturus’ commitment to the development of novel RNA therapeutics has led to collaborations with Janssen Pharmaceuticals, Inc., part of the Janssen Pharmaceutical Companies of Johnson & Johnson, Ultragenyx Pharmaceutical, Inc., Takeda Pharmaceutical Company Limited, CureVac AG, Synthetic Genomics Inc., Duke-NUS Medical School, and the Cystic Fibrosis Foundation. For more information visit www.ArcturusRx.com. In addition, please connect with us on Twitter and LinkedIn.

SOURCE: Arcturus Therapeutics

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