Arcturus’ RNA Therapy for CF Shows Promise in Preclinical Studies, Company Announces

Arcturus Therapeutics’ RNA-based approach enables the production of healthy CFTR — the protein whose dysfunction is key in cystic fibrosis (CF) — and can be effectively delivered into lung cells, according to the company’s preclinical results.

This CF treatment candidate is being developed in collaboration with Cystic Fibrosis Foundation Therapeutics, the drug discovery and development affiliate of the Cystic Fibrosis Foundation.

CF is caused by mutations in the CFTR gene, leading to the malfunction of the CFTR protein. This results in the production of thick, sticky mucus in the lungs, pancreas, liver, and intestine, among other symptoms.

San Diego-based Arcturus is using its LUNAR lipid (fat)-mediated delivery technology to deliver messenger RNA molecules, which contain information to make functional CFTR proteins, into target cells.

Arcturus’ LUNAR approach for CF is intended to treat all patients, regardless of their specific mutations.

The company’s preclinical research in cells and mice, “LUNAR-CF, a mRNA Replacement Therapy for Cystic Fibrosis,” was presented at the Cystic Fibrosis Foundation Research Conference, recently held in Jackson Hole, Wyoming.

Among the findings presented, researchers revealed new human mRNAs that yielded higher levels of functional CFTR protein, as well as evidence of efficient mRNA delivery into lung epithelial cells using LUNAR-CF.

Additionally, the scientists showed that the LUNAR technology allowed for efficient nebulized, or inhaled, delivery and shields the mRNA from degradation in CF sputum.

Based on these positive results, the CF Foundation made an undisclosed payment to Arcturus to advance the development of LUNAR-CF. The two organizations also agreed to expand their research agreement and amend the milestone criteria to fine-tune the path ahead before starting clinical trials. Arcturus will now accelerate the development of an mRNA-based therapy for CF using LUNAR.

“We are pleased to report that LUNAR-CF has advanced through key milestones with the support of the CF Foundation,” Joseph Payne, Arcturus’ president and CEO, said in a press release. “This enhanced agreement underscores the CF Foundation’s continued commitment to our cystic fibrosis development program.”

“Our goal is to develop a transformative therapeutic that has the potential to treat a broad population of cystic fibrosis patients, regardless of mutation type,” Payne added, noting the company’s confidence in successfully translating its lead treatment candidate into the clinic.

In 2014, Arcturus reported the successful in vitro transfer of the CFTR mRNA into cells.