The therapy will use the company’s LUNAR delivery technology that comprises a library of more than 150 proprietary fatty molecules specifically designed to deliver RNA-based therapies to a particular cell type of interest, according to Arcturus.
In the case of ARCT-032, LUNAR will be employed to deliver an aerosolized form of the therapy, which contains the messenger RNA (mRNA) that provides instructions to create the CFTR protein, directly to lung cells. By enabling these cells to produce CFTR, which is either absent or dysfunctional in CF patients, ARCT-032 is designed to restore the protein’s activity in the lungs and alleviate CF symptoms.
“We are pleased to have advanced ARCT-032 as a novel mRNA-based development candidate for CF lung disease,” Pad Chivukula, PhD, chief scientific officer and chief operating officer of Arcturus, said in a press release.
“We believe that ARCT-032 has the potential to address the root cause of CF lung disease, which is caused by the defective, or missing, CFTR protein,” he added.
The company’s decision to move forward with ARCT-032’s development program was supported by data from preclinical studies conducted in several animal models of disease.
Data from some of these studies showed the LUNAR platform could be used to successfully deliver CFTR mRNA to cells lining the inside of the airways in a CF mouse model following nasal inhalation. Treatment was also found to restore CFTR activity in the animals’ lung cells, where the protein normally works as a channel to transport water and chloride.
If successfully developed, ARCT-032 may potentially become a RNA-based replacement therapy that can be used to treat all CF patients, regardless of the particular type of genetic mutation they carry, according to the company. This is because ARCT-032 focuses on restoring the production of functional CFTR rather than repairing protein defects caused by specific mutations, which is the case with other CF treatments.
“While there has been progress with the recent approval of drugs for CF, many patients remain underserved,” Chivukula said. “ARCT-032 has the potential to provide benefit to all CF patients, regardless of their underlying genetic mutations.”
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