The Cystic Fibrosis Foundation is investing up to $9 million in additional funds in Arcturus Therapeutics to test ARCT-032, an inhaled messenger RNA (mRNA) therapeutic candidate 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.

This agreement extends a prior research program, initiated in 2017, which has resulted in the advancement of ARCT-032 into ongoing clinical studies.

ARCT-032 is designed to deliver the mRNA template to the lung cells to produce functional cystic fibrosis transmembrane conductance regulator (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,” says JP Clancy, MD, senior vice president of clinical research at the foundation, in a release. “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 cystic fibrosis 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.

“We are thrilled to extend our productive relationship with the Cystic Fibrosis Foundation, and we are grateful for their meaningful financial support,” says Pad Chivukula, PhD, chief scientific officer of Arcturus, in a release. “ARCT-032 has the potential to express fully functional CFTR protein in the lung, thereby addressing the root cause of cystic fibrosis. The resources and valuable expertise provided by the CF Foundation will support the continued clinical development of ARCT-032, including the completion of a phase 1b study that we plan to initiate imminently in adults living with cystic fibrosis.”

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

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