Patricia Inácio, PhD, science writer —

Long-term treatment with Kalydeco (ivacaftor) is well-tolerated and efficient, with improvements in sweat chloride concentration, growth, and pancreas function maintained over 84 weeks in 2-to-5-year-old children with cystic fibrosis (CF), results from the Phase 3 KLIMB extension study show. Trial findings were published in the study, “An…

A mouse model carrying the most common mutation in the CFTR gene (the gene defective in patients with cystic fibrosis) shows disturbances in genes regulating the sleep cycle known as the circadian clock. The study “Dysregulation of Circadian Rhythm Gene Expression in Cystic Fibrosis Mice” was published in the…

Poor oxygen conditions, a common trait in the lungs of cystic fibrosis (CF) patients, may help Pseudomonas aeruginosa bacteria outcompete other infectious agents, namely Staphylococcus aureus, a study suggests. The study, “Anaerobiosis influences virulence properties of Pseudomonas aeruginosa cystic fibrosis isolates and the interaction with Staphylococcus aureus,” was…

The RESTORE-CF clinical trial investigating Translate Bio’s candidate therapy MRT5005 has completed the single-ascending dose regimen in patients with cystic fibrosis (CF), the company announced. The ongoing Phase 1/2 trial (NCT03375047) is testing the safety and efficacy of single and multiple escalating doses of nebulized MRT5005, compared with each other and…

Using the gene editing tool CRISPR-Cas9 to specifically target the lung of a developing fetus, researchers were able to correct a mutation associated with a human lung disease that is fatal after birth, a study in mice shows. These findings highlight the potential of gene editing strategies to correct mutations…

Treating an animal model of cystic fibrosis (CF) with Kalydeco (ivacaftor) during embryonic development and after birth revealed that early treatment improved pancreatic function, delayed lung disease, and reduced infections, a study suggests. The study, titled “In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model…

Pseudomonas aeruginosa, the most common bacteria found in cystic fibrosis (CF) patients, uses a virus to go unnoticed by the host immune system, a new study shows. Treatment with a vaccine designed to help the immune system detect the virus was found to significantly reduce P. aeruginosa’s ability to infect mice,…

Removing a key metabolite called pyruvate from Pseudomonas aeruginosa and Staphylococcus aureus bacteria biofilms — two of the most common bacteria found in cystic fibrosis (CF) patients — could help boost the effectiveness of antibiotics, a study reports. The study, “Pyruvate-depleting conditions induce biofilm dispersion and enhance…

A new study shows that bacteriophages, viruses that naturally infect bacteria, can be used to fight Pseudomonas aeruginosa, the most common bacteria found in cystic fibrosis (CF) patients. Using a freshwater fish model of CF, researchers saw that a combination of bacteriophages and antibiotics is effective against the disease. The…

Cystic fibrosis (CF) patients in Australia age 12 and older, who have two copies of the F508del mutation in the CFTR gene (a copy inherited from each parent), now can be treated with Vertex Pharmaceuticals’ Symdeko (tezacaftor/ivacaftor and ivacaftor). The treatment also was approved for any mutation in the CFTR gene that responds to Symdeko based on lab…