Preclinical Messenger RNA Data of New LUNAR Cystic Fibrosis Therapy Shows Promise

Preclinical Messenger RNA Data of New LUNAR Cystic Fibrosis Therapy Shows Promise

LUNAR Cystic Fibrosis TherapyDuring the 2nd International mRNA Health Conference being held on November 11-12, 2014 in Cambridge, MA, Arcturus Therapeutics, Inc., a leading company in RNA therapies for rare diseases, will present the poster (#13), “Potent Delivery of LUNAR™ Nanoparticles Containing Synthetic mRNA for Therapeutic Protein Expression in Non-Human primates.” The new findings could eventually offer those with Cystic Fibrosis a novel therapy for treating the disease.

Founded in 2013 and located in San Diego, Arcturus Therapeutics, Inc. is a pioneer in the research and development of technologies for the delivery of RNA molecules. The company has developed a new, effective and safe RNA Therapeutics platform called LUNARTM, a proprietary lipid-enabled delivery system for RNA medicines including small interfering RNA, messenger RNA, antisense, and microRNA oligotherapeutics.

In this study, the researchers injected one low dose (0.3 mg/kg) of LUNAR™ delivery of human erythropoietin messenger RNA in non-human primates (N = 3) and observed an increase of 1000-fold in the production of the protein erythropoietin. During the study none of the non-human primates showed adverse effects due to the treatment, demonstrating that LUNAR delivery of messenger RNA was well tolerated.  In the poster presentation, the researchers will also present preclinical results from the studies using rodent models treated with therapeutic human messenger RNAs for alpha-1 antitrypsin (AAT), factor IX, and erythropoietin.  They found that the delivery of the chemically modified messenger RNA in LUNAR™ for these proteins resulted in rapid expression of the proteins after 2 hours and with the  effect lasting for 24 hours after administration. Additionally, they will show the results from the in vitro cell culture studies with cystic fibrosis transmembrane conductance (CFTR) and tumor protein p53 (p53) messenger RNA. All these data demonstrate the wide potential of applications of this new therapeutic strategy.

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“Messenger RNA offers a powerful way to modulate gene expression within a cell, thereby providing a novel therapeutic approach to treat diseases that were previously inaccessible or relied on expensive and complex protein replacement therapies. We are thrilled to be presenting our messenger RNA data in rodents and non-human primates, showing impressive production of multiple human therapeutic proteins with a large safety margin due to our proprietary LUNAR delivery technology,” said Dr. Pad Chivukula, CSO and COO of Arcturus Therapeutics, in the press release. “UNA chemistry and LUNAR delivery provide the extraordinary flexibility to effectively modulate gene expression in BOTH directions — increasing protein expression via messenger RNA and/or decreasing gene activity via small interfering RNA. Arcturus is in a unique position having proven successful RNA delivery capability in non-human primates for both messenger RNA and siRNA.” concluded Dr. Pad Chivukul.


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