Molecular therapy : the journal of the American Society of Gene TherapyDec 25, 2025

LUNAR® nanoparticle delivery of CFTR mRNA restores channel function and improves mucus clearance in human and ferret cystic fibrosis airways

mRNA Technology Weekly Brief ↗PubMed ↗DOI ↗

Updated Jun 27, 2026

Abstract

Essence

LUNAR-CFTR lipid nanoparticles delivered CFTR mRNA and improved cystic fibrosis airway function in cell and ferret models.

Evidence

Preclinical studies in primary human and ferret airway epithelia, mucus-laden CF ferret airways, and transgenic ferrets showed CFTR mRNA delivery, restored ion transport, and a 3-fold mean mucociliary-clearance improvement after one dose.

Caveat

The evidence is preclinical, with single-dose ferret outcomes and in vitro airway models rather than human clinical efficacy or durability data.

Simplified

Lipid nanoparticle (LNP)-based delivery of CFTR mRNA holds promise for treating pulmonary manifestations of cystic fibrosis (CF). LUNAR-CFTR is a novel proprietary lipid nanoparticle (LNP) encapsulating codon-optimized human CFTR mRNA for CFTR replacement therapy. In this study, we examined the potential of LUNAR-CFTR for delivering mRNA and restoring CFTR function in primary human and ferret airway epithelia and mucus-laden CF ferret airways. LUNAR effectively delivered CFTR, Cre, and tdTomato mRNAs into multiple airway epithelial cell types of humans and ferrets in vitro and transgenic ferrets in vivo. The LUNAR-CFTR was able to restore ion transport in polarized CF human bronchial epithelia to levels comparable to those achieved with elexacaftor/tezacaftor/ivacaftor. Mucociliary clearance (MCC) assays in CF ferrets further showed that a single dose of LUNAR-mediated human CFTR mRNA delivery improved mean MCC values by 3-fold. The improved MCC was in line with an increase in CFTR-mediated transepithelial anion transport from the CF ferret trachea transfected with LUNAR-CFTR in vivo. These findings underscore the potential of LUNAR LNP as a robust vehicle for delivering CFTR-encoding therapeutic mRNA into airway epithelial cells and underscore the utility of CF ferrets as a reliable tool to evaluate the efficiency of gene therapy in mucus-laden airways.

Full Text

Full text is available at the source.

View Full Text ↗

Featured in

mRNA TechnologyIssue #17

mRNA flu vaccines work in newborn primates — and an AI-designed herpes vaccine shows promise in mice

↗

LUNAR® nanoparticle delivery of CFTR mRNA restores channel function and improves mucus clearance in human and ferret cystic fibrosis airways

Abstract

Full Text

Featured in

You found one interesting study. We’ll send the next 7.

what lands in your inbox each week:

Recent issues from the mrna technology brief