Phase 1 Trial of ARCT-032 RNA Therapy for CF Cleared to Start

Experimental treatment works to restore functional CFTR production

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

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A Phase 1 trial of ARCT-032, an inhaled experimental treatment for cystic fibrosis (CF), has been cleared to start in New Zealand.

Developed by Arcturus Therapeutics, ARCT-032 contains the messenger RNA (mRNA) that provides instructions for lung cells to produce the CFTR protein, restoring its activity. Mutations in the CFTR gene, leading to the protein’s impairment or absence, are the root cause of CF.

“Arcturus continues to progress ARCT-032, a potential mRNA medicine for people with cystic fibrosis regardless of their underlying mutation type, through the approval of a [clinical trial application] to proceed into First-in-Human studies,” said Joseph Payne, the company’s CEO, in a press release.

The CFTR protein regulates the movement of chloride ions (negatively charged salt particles) and water into and out of cells. This helps produce mucus that several organs, including the lungs and digestive tract, need to function.

Mutations in the CFTR gene result in a thick mucus that clogs the airways, causing respiratory difficulties and recurrent lung infections.

Currently available CFTR modulator therapies are designed to increase the protein’s function and help ease CF symptoms. Their effectiveness depends on the underlying mutations, however.

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ARCT-032 works to restore the production of functional CFTR instead of repairing defects that are caused by specific mutations. This means, if it’s successful, ARCT-032 can treat all CF patients regardless of their mutation, according to the company.

The Phase 1 trial is the first time ARCT-032 will be tested in humans, Arcturus noted.

“Preclinical data shared at the recent North American Cystic Fibrosis Conference demonstrated robust expression and functional restoration of CFTR in human bronchial epithelial cells from CF donors, providing additional support for the advancement of ARCT-032 into clinical development,” Payne said.

The therapy utilizes Arcturus’ LUNAR proprietary technology, which makes up a library of more than 250 fatty molecules that can be specifically designed to deliver mRNA to a target cell type, according to the company.

“We believe the advantages driven by our proprietary LUNAR delivery technology, advanced mRNA purification processes, and manufacturing know-how may allow ARCT-032 to restore healthy CFTR protein in the lungs of people with CF, including those that currently do not have effective treatment options,” Payne said.

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