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May 30, 2017

Arcturus Partners with CFFT on mRNA Therapeutic for Cystic Fibrosis

  • Arcturus Therapeutics said today it will receive $3 million from the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation under a research agreement designed to advancer the company’s novel messenger RNA (mRNA) therapeutic LUNAR-CF.

    The two-year agreement with Cystic Fibrosis Foundation Therapeutics Inc. (CFFT) is designed to support discovery and development of mRNA therapeutics designed to enable cells of CF patients to restore the function of their cystic fibrosis transmembrane conductance regulator (CFTR) gene in their lungs.

    LUNAR-CF is a cystic fibrosis treatment formulated with Arcturus' Lipid-enabled and Unlocked Nucleomonomer Agent modified RNA (LUNAR™) nanoparticle delivery technology. LUNAR is designed to transport all types of RNA medicines to target cells, and according to Arcturus has shown excellent safety and potency in multiple animal species.

    LUNAR nanoparticles encapsulate RNA and deliver it straight into the cell via a process called endocytosis, during which they fuse with the cell membrane and enter the cell. The lipid nanoparticles are rapidly degraded, leaving the RNA inside the cell where it can work. The nanoparticles can be modulated to target specific cell types.

    According to Arcturus, pre-clinical in vivo studies have shown that LUNAR technology can prevent potency drift with repeat RNA medicine dosing. Unlike viral vectors, LUNAR nanoparticles do not accumulate inside targeted cells, are not immunogenic and cannot cause insertional mutagenesis.

    Under the collaboration with CFFT, LUNAR will be advanced and optimized for pulmonary administration, enabling the development of novel nucleic acid based therapeutics for a variety of respiratory diseases, the company said.

    “Arcturus is developing a potentially transformative broad-spectrum mRNA therapeutic. If successful, it has the potential to treat all CF patients—more than 1,700 different genetic variations of the disease,” Arcturus President and CEO Joseph E. Payne said in a statement.

    Arcturus has also applied LUNAR to develop treatment candidates for transthyretin-mediated amyloidosis or ATTR (LUNAR-TTR); ornithine transcarbamylase (OTC) deficiency (LUNAR-OTC); and an unspecified genetic liver disease that affects infants and children (LUNAR-GI).

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