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Researchers have developed nanoparticles that can deliver gene-editing therapeutics to correct genetic defects in the lungs of people with cystic fibrosis (CF). The nanoparticles were optimized using cell-based models to penetrate the thick airway mucus seen in CF. Pretreating patients with the approved mucus-clearing agent Pulmozyme (dornase alfa)…

A University of Iowa biomedical researcher was awarded the Yergin-New International Prize for his work in understanding cystic fibrosis (CF) — with discoveries that helped reveal the mechanisms underlying the genetic condition, and led to the development of a new animal model of CF that captures key features of…

SPL84, an investigational inhalation therapy designed to treat cystic fibrosis (CF) caused by a specific mutation called 3849+10 kb C-to-T, was well tolerated in a Phase 2 clinical trial. Trial data also suggest that most CF patients treated with SPL84 experienced improvements in a measure of lung function, according…

Researchers have identified three groups of symptoms with interconnected severity — respiratory-energy, mood-gastrointestinal irritability, and pain-gastrointestinal abnormal motility — in people with cystic fibrosis (CF). The severity in each group was associated with clinical and demographic factors, including lung function, treatment with standard CFTR modulators, age, ethnicity, and…

The first participants have been dosed in a clinical trial that’s testing some of Sionna Therapeutics’ investigational treatments for cystic fibrosis (CF). The Phase 1 study (NCT07035990) is testing SION-451 with either SION-2222 or SION-109 in healthy volunteers, with the main goal being to evaluate the safety…

Northwestern University scientists developed a wearable patch that could help cystic fibrosis (CF) patients manage their disease from home. A study showed that the patch, developed in collaboration with scientists at Epicore Biosystems, measured sweat chloride in test participants as effectively as the gold standard method for sweat chloride assessment…

The Food and Drug Administration (FDA) placed the U.S. portion of a Phase 2b trial of BX004, a treatment for Pseudomonas aeruginosa infection in people with cystic fibrosis (CF), on clinical hold because of questions about a third-party drug delivery device. The treatment’s developer, Biomx, believes the FDA’s…

In people with cystic fibrosis (CF), treatment with Kaftrio (elexacaftor, tezacaftor, and ivacaftor) may reduce the risk of CF-related liver disease (CFLD) progression, according to a real-world study in France. Rates of severe liver complications were significantly lower among people with CF who were followed during the period after Kaftrio …

Interim results from the first nine participants in a Phase 2 clinical trial testing Arcturus Therapeutics’ experimental inhaled therapy ARCT-032 in adults with cystic fibrosis (CF) are expected next month. The U.S.-based study, called LunairCF (NCT06747858), is still recruiting CF patients who aren’t eligible for CFTR…

A new noninvasive prenatal test for cystic fibrosis (CF) and three other inherited conditions, launched by Natera, uses a sample of the mother’s blood to screen fetal DNA. The test, called Fetal Focus, may be particularly useful when the baby’s biological father isn’t available for genetic testing to…