RARE Daily

Cystic Fibrosis Foundation Commits Up to $15 Million to Arcturus for mRNA Therapies

August 2, 2019

The Cystic Fibrosis Foundation has increased its commitment to Arcturus Therapeutics to $15 million in conjunction with an amended agreement to advance Arcturus’ novel messenger RNA therapeutic, LUNAR-CF.

The goal of the multi-year program is to create mRNA therapies to treat people with cystic fibrosis (CF), develop methods to deliver RNA components to cells in the lung and file an Investigational New Drug (IND) application for a therapeutic candidate. The new commitment continues a two-year agreement between the parties entered into in 2017.

Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It causes mucus buildup that can clog some organs in the body, especially the lungs and pancreas. In the lungs, it can make breathing difficult. It also causes bacteria to get stuck in airways, making CF patients more prone to inflammation and infections, leading to permanent lung damage and fibrosis. 

“We are pleased with the progress we have made in our agreement with the CF Foundation, including 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,” said Joseph Payne, president and CEO of Arcturus Therapeutics. 

LUNAR-CF, Arcturus’ first inhaled mRNA therapeutic targeting the lung, represents a novel approach to treat CF.  It is an mRNA replacement therapy designed to enable CFTR-deficient patients to naturally produce healthy functional CFTR in their own lung cells.  Arcturus plans to submit an application to market the drug to the U.S. Food and Drug Administration in the second half of 2020. 

Photo: Joseph Payne, president and CEO of Arcturus Therapeutics. 

Author: Rare Daily Staff

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