Lindsey Shapiro, PhD, science writer —

Lung transplant rates among people with cystic fibrosis (CF) in the U.S. have declined dramatically since the approval of Trikafta (elexacaftor/tezacaftor/ivacaftor), a study found. However, almost half of patients who received transplants in recent years were on Trikafta before their procedures. Moreover, a notable proportion of CF patients…

Nearly two-thirds of cystic fibrosis (CF) patients in a study showed signs of type 2 inflammation, which was associated with worse lung function, more infections, and a higher risk of death. This form of inflammation, most commonly linked to allergic conditions, was reduced somewhat after treatment with CFTR…

Additional mutations in the SLC26A9 gene among cystic fibrosis (CF) patients with two copies of F508del — the most common CF-causing mutation in the CFTR gene — were associated with more rapidly declining lung function. These findings from a study further buttress the idea that variation in genes other…

A lung-targeted gene-editing therapy designed using technology from ReCode Therapeutics was able to successfully correct a disease-causing CFTR gene mutation in lung cells derived from cystic fibrosis (CF) patients, according to a recent proof-of-concept study. The therapy, developed using ReCode’s Selective Organ Targeting (SORT) lipid nanoparticles (LNPs), was…

Exposure in utero to the cystic fibrosis (CF) medication Trikafta (elexacaftor/tezacaftor/ivacaftor) didn’t appear to negatively affect organ development in fetal rats whose mothers were given it, but it did change the activity of some genes, a study finds. While the functional impacts of the changes remain to be…

A lung transplant can be lifesaving for children with cystic fibrosis (CF) admitted for respiratory failure to a pediatric intensive care unit (PICU). Only 35% of these young patients went on to be discharged without a transplant, according to an analysis of children with CF treated at a French…

Researchers have developed a device that could improve gene therapy delivery to the lungs for people with genetic conditions such as cystic fibrosis (CF). In a series of preclinical experiments, the research team showed that its device could deliver a therapeutic payload to cells better than a standard inhaler…

In a recent study, scientists described the process through which they discovered GLPG2737, an investigational CFTR modulator considered by Galapagos and AbbVie to be a possible treatment for cystic fibrosis (CF). The molecule was found in early work to have an additive effect when combined with other types…

Treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor) led to gains in body mass among cystic fibrosis (CF) patients that were largely driven by increases in various types of fat and low-density muscles, a study found. High-density muscle, which is stronger than its lower-density counterparts, didn’t change with the treatment, sold as…

In lab studies, researchers have identified rare mutations in the CFTR gene — which are the cause of cystic fibrosis (CF) — that are not currently eligible for CFTR modulator therapies but might be treatment responsive. The researchers hope the findings will provide guidance to developer Vertex Pharmaceuticals…