RARE Daily

CF Foundation Invests Up to $9 Million in Arcturus to Develop a Messenger RNA Therapy

September 27, 2023

Rare Daily Staff

The Cystic Fibrosis Foundation is investing up to $9 million in additional funds in Arcturus Therapeutics to test an inhaled messenger RNA (mRNA) therapy that could treat the underlying cause of cystic fibrosis in all people living with the disease, including those with two copies of rare and nonsense mutations.

Cystic fibrosis (CF) is a rare, progressive, genetic disease that affects the lungs, pancreas, and other organs. In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional. When the protein is not working correctly, it’s unable to help move chloride—a component of salt—to the cell surface. Without the chloride to attract water to the cell surface, the mucus in various organs becomes thick and sticky. In the lungs, the mucus clogs the airways and traps germs, like bacteria, leading to infections, inflammation, respiratory failure, and other complications.

Arcturus’ mRNA therapy is designed to deliver the mRNA template to the lung cells to produce functional CFTR protein. The mRNA would be delivered to cells within a lipid nanoparticle, a chemically coated fat-like particle designed to help the mRNA enter cells more easily.

“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 J.P. Clancy, senior vice president of clinical research at the Foundation. “Delivering the therapy to the correct cells is a significant challenge, and we are excited by the progress Arcturus is making in this area.”

Dosing has been completed in a phase 1a clinical trial of Arcturus’ RNA therapy in healthy people in New Zealand, and a phase 1b trial in people with CF in New Zealand has been approved. The Foundation’s funding will support this trial, which is planned for the fourth quarter of 2023. Arcturus also plans to use the funding for additional research prior to a planned phase 2 trial in 2024.

The additional $9 million in funding combined with the $15.6 million already provided to Arcturus brings our total commitment to nearly $25 million. The new investment comes from the $500 million Path to a Cure, an ambitious research initiative to accelerate treatments for everyone with CF and ultimately deliver a cure. The Foundation is funding research into mRNA therapies because of their potential to treat all people with CF.

Photo: J.P. Clancy, senior vice president of clinical research at the Foundation

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