The CF Foundation Awards Arcturus Therapeutics Up to $15M for RNA Therapy

The Cystic Fibrosis Foundation has increased its award to Arcturus Therapeutics to up to $15 million to develop a mutation-agnostic therapy for people with cystic fibrosis.

| 2 min read

The Cystic Fibrosis Foundation has increased its award to Arcturus Therapeutics to up to $15 million to advance a potential therapy that could help all people with cystic fibrosis produce a normal cystic fibrosis transmembrane conductance regulator (CFTR) protein, regardless of their mutation.

Arcturus is developing a way to deliver normal messenger RNA (mRNA) into lung cells so that the cells would have the correct instructions to produce healthy CFTR protein. In cystic fibrosis, mRNA carries faulty instructions to the protein-making machinery of the cell, which results in the production of either a dysfunctional CFTR protein or no CFTR protein at all.

The goal of Arcturus' multi-year LUNAR-CF program is to produce and advance an Investigational New Drug (IND) application for both the novel mRNA therapy and a special lipid

 delivery system that would allow the mRNA to enter lung epithelial cells. The lipid coating forms a liposome that fuses with the outer membrane of cells -- like two soap bubbles merging -- and would allow mRNA from the liposome to transfer to the inside of the cell. The technology is still undergoing preclinical testing.

For more information, see the Arcturus release.

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