The Cystic Fibrosis Foundation has announced an investment of up to $9 million in Arcturus Therapeutics to test an inhaled messenger RNA (mRNA) therapy with potential to treat the underlying cause of cystic fibrosis.

The mRNA therapy is designed to deliver the mRNA template to the lung cells to produce functional cystic fibrosis transmembrane conductance regulator (CFTR) protein. Dosing has been completed in a Phase 1a clinical trial in healthy people in New Zealand, and a Phase 1b trial in people with CF in New Zealand has been approved. Arcturus also plans to use the funding for additional research prior to a planned Phase 2 trial in 2024. The latest commitment brings the foundation’s total investment in Arcturus to nearly $25 million.

“Messenger RNA therapy offers a way to treat all people with CF, especially those who can’t tolerate or don’t respond to modulator treatment,” said CF Foundation senior vice president of clinical research J.P. Clancy. “Delivering the therapy to the correct cells is a significant challenge, and we are excited by the progress Arcturus is making in this area.”

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