Arcturus Therapeutics

Arcturus is collaborating with Cystic Fibrosis Foundation Therapeutics, Inc. to develop a messenger RNA (mRNA) medicine to treat cystic fibrosis.


Cystic fibrosis

Cystic fibrosis is a common life-threatening genetic disease in the United States. The disease is caused by over 2,000 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. There are over 70,000 patients world-wide.

There are no FDA-approved drugs that can treat all 2,000 CFTR mutations. The FDA has approved three CFTR modulator therapies, Kalydeco®, Orkambi® and Symdeko® (Vertex Pharmaceuticals Inc.), to treat fewer than 40 cystic fibrosis-causing mutations. These drugs do not treat the underlying genetic cause of cystic fibrosis.

We are developing an mRNA therapeutic using our LUNAR delivery platform to deliver normal CFTR mRNA into airway epithelial cells.

LUNAR-CF solution

This allows airway cells to produce functional CFTR protein using their normal cellular machinery. This approach will be the first to treat the underlying defect that causes cystic fibrosisdysfunctional or absent CFTR proteinin all patients, regardless of mutation type.

We have completed preclinical proof of concept studies, demonstrating that LUNAR is able to deliver mRNA efficiently into lung epithelial cells in animals and is compatible with nebulization. In cell-based assays, we have also demonstrated that our lead CFTR mRNA has improved functional activity and longer duration of protein expression compared to a reference CFTR mRNA.

We aim to achieve functional correction of rare diseases using our proprietary LUNAR-delivered mRNA therapeutic candidates.