Arcturus Therapeutics

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

LUNAR-CF

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.

Our 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.

LUNAR-RLD

The LUNAR-RLD program is an internal research program focused on target validation of multiple pipeline LUNAR-mRNA program candidates. To accomplish this goal, we have prioritized a list of rare liver diseases. All have significant unmet medical needs and few treatment options are available to patients. Arcturus will use its LUNAR delivery platform to deliver normal mRNA into target cells of interest, allowing these cells to produce functional protein using their native translational machinery and protein trafficking pathways.

Arcturus has identified a lead small interfering RNA (siRNA) compound to treat transthyretin-mediated amyloidosis, a genetic disease that affects approximately 1 in 100,000 throughout the U.S. and Europe.

LUNAR-TTR

Transthyretin-mediated amyloidosis

Transthyretin-mediated amyloidosis is an inherited genetic disease caused by over 125 different mutations in the transthyretin (TTR) gene. Patients with transthyretin-mediated amyloidosis can have severe heart and neurological problems including heart failure, muscle weakness and wasting, and weight loss.

There are no FDA-approved drugs to treat transthyretin-mediated amyloidosis. Patients are currently treated with supportive care and with liver and heart transplants.

Our novel small siRNA therapeutic candidate to treat the genetic cause of transthyretin-mediated amyloidosis, is available for out licensing.

Our LUNAR-TTR solution

siRNA can be used to target any mutation in the transthyretin (TTR) gene. Using our LUNAR delivery platform, we have achieved proof of concept in non-human primates and are actively pursuing partnership opportunities to complete investigational new drug (IND)-enabling studies.

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