Steve Bryson, PhD,  science writer—

Researchers have identified a new class of CFTR correctors to treat cystic fibrosis (CF) that enhance the potency of currently available CFTR modulator therapies, a study shows. Data reveal that these compounds demonstrated high potency in airway cells derived from CF patients. The study, “Novel tricyclic…

A single dose of 4D Molecular Therapeutics’ 4D-710, an inhaled gene therapy candidate for adults with cystic fibrosis (CF), improved or stabilized lung function in three patients in a Phase 1/2 clinical trial. Nine to 12 months of follow-up data showed it led to meaningful gains in quality…

People with cystic fibrosis (CF) who frequently use healthcare resources accounted for almost one-half of total healthcare costs, according to a new Canadian study. Becoming a frequent high-cost CF healthcare user was linked to severe lung impairment, lung transplant, and liver cirrhosis (a chronic liver disease) with high blood…

Beyond improving lung function, Orkambi (ivacaftor/lumacaftor) has potent anti-inflammatory benefits that may limit immune-related lung damage in people with cystic fibrosis (CF), according to a real-world study. Researchers found that one year of treatment significantly reduced the levels of pro-inflammatory signaling molecules in the bloodstream and airways of…

The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for children with cystic fibrosis (CF) as young as one month old, who have at least one gating mutation in their CFTR gene that is responsive to the oral treatment. The agency’s approval was supported by data…

A rare cell type that lines the airways, called pulmonary ionocytes, was successfully generated from stem cells derived from people with cystic fibrosis (CF), a study reports. Among all the cell types that line the airways, the recently discovered ionocytes, which make up about 1% of cells, have the…

Lung function decline in adults with stable cystic fibrosis (CF) was tied to high numbers of mature low-density neutrophils, a white blood cell subtype associated with inflammatory disorders, a study suggests. While previous research showed that low-density neutrophils in CF children correlated with better lung function, the changes found…

Researchers are developing new inhalable nanoparticles designed to deliver RNA-based gene-editing therapeutics directly to the lungs, a study reports. Now shown to be effective in mice, the ultimate goal is to develop inhaled therapeutics to correct genetic defects in conditions affecting the lungs, such as cystic fibrosis (CF). “This…

Small changes in the shape of CFTR, the protein defective or absent in people with cystic fibrosis (CF), opened its gate outside the cell to allow the flow of chloride ions, a study reported. CF-causing mutations that suppress CFTR function disrupt these long-distance shape changes. By comparison, CFTR potentiator…

Trikafta increased the production of the faulty CFTR protein in cystic fibrosis (CF) patients, but it remained in an immature state, a study suggests. These findings may shed light on the wide variability in response rates in CF patients and caution that immature CFTR protein may prevent sustained…