Steve Bryson, PhD,  science writer—

Scientists at the Broad Institute of MIT and Harvard have created a gene editing technology that may be able to insert or substitute entire genes in human cells, which could pave the way for new gene therapies for diseases such as cystic fibrosis (CF). The approach could efficiently insert…

CF AMR Syndicate, a U.K. research support group, has awarded BioVersys up to £500,000 ($636,000) to develop small molecules targeting hard-to-treat nontuberculous mycobacteria (NTM) infections in people with cystic fibrosis (CF). Established in 2019 as a cross-sector (e.g., public-private) initiative with the Cystic Fibrosis Trust to…

A single dose of 4D Molecular Therapeutics’ inhaled gene therapy candidate 4D-710 improved or stabilized lung function in adults with cystic fibrosis (CF), according to interim data from a Phase 1/2 clinical trial. Additional data from the AEROW (NCT05248230) study, which is evaluating the therapy in CF…

A miniaturized wearable device is able to measure chloride levels in sweat, the gold standard tool for diagnosing cystic fibrosis (CF). In infants with CF, tests showed the device’s accuracy matched standard methods without the need for trained personnel in hospital settings. It also measured sweat levels of zinc…

Newborn screening (NBS) in Switzerland correctly identified 96% of cystic fibrosis (CF) cases over an 11-year period, a study found. False negatives, instances where tests are negative but an infant has CF, occurred in 10 out of 272 diagnosed cases. Still, the program did not achieve a positive predictive…

After starting Trikafta, people with cystic fibrosis (CF) significantly reduced their use of other CF-related medications, suggesting that many people with CF on Trikafta “may prioritize reducing treatment burden over maximizing lung function,” researchers said. Still, lung function continued to decline after Trikafta initiation, they reported in a real-world…

Pulmonary ionocytes, a rare airway cell type that produces high levels of CFTR — the protein that’s defective in cystic fibrosis (CF) — were found to regulate the acidity of the liquid coating the surface of the airways, a cell-based study showed. Results also showed that club cells, another…

The European Commission (EC) has expanded the approval of Kalydeco (ivacaftor) for the treatment of infants with cystic fibrosis (CF) as young as 1 month with at least one CFTR gating mutation. The approval makes Kalydeco, marketed by Vertex Pharmaceuticals, the first and only CFTR modulator…

A grant of $500,000 in Australian dollars (around $326,000) will support researchers there in the development of antibiotics formulated with liquid crystalline nanoparticles (LCNPs), the better to eliminate hard-to-treat lung infections in people with cystic fibrosis (CF). “By overcoming the processes that cause drug resistance and uncontrollable infection, this…

Elevated levels of the pro-inflammatory signaling protein galectin-3 in the bloodstream of children with clinically stable cystic fibrosis (CF) significantly correlated with worse lung function, according to a study. The findings suggest galectin-3 may serve as a noninvasive biomarker to predict lung disease outcomes in children with CF, the…