José Lopes, PhD, managing science editor —

Translate Bio is going to focus on the development of potential therapies to treat pulmonary diseases, including its lead candidate, MRT5005, targeting the underlying cause of cystic fibrosis (CF), the company announced. MRT5005 is designed to restore the levels of working cystic fibrosis transmembrane conductance regulator (CFTR) protein and reverse…

A young girl with cystic fibrosis (CF) and acute recurrent pancreatitis was able to eat some food normally and experienced relief of her abdominal symptoms after undergoing a complete removal of her pancreas and a transplant of pancreatic cells into her liver, according to a case report. At…

Cystic fibrosis (CF) patients treated with Kalydeco (ivacaftor) who have the G551D mutation reported better health-related quality of life (HRQoL) and symptom relief than those on standard treatment who carry the F508del mutation in the CFTR gene, according to a real-world study. The research, “Patient-reported…

The European Medicines Agency (EMA) granted orphan drug status to AR-501, a treatment candidate for lung infections in people with cystic fibrosis (CF). AR-501, developed by Aridis Pharmaceuticals and also known as Panaecin, is an inhaled formulation of gallium citrate intended as a weekly and self-administered therapy delivered…

Approved and investigative cystic fibrosis (CF) treatments known as CFTR potentiators work by binding to the same flexible protein spot — a place that acts like a hinge — and keeping it open, according to a study whose findings may lead to more effective therapies. The research, “Structural…

Canadian patients with cystic fibrosis (CF) have better respiratory function and higher body mass index (BMI) than French patients, although they have higher blood glucose levels and a greater incidence of CF-related diabetes (CFRD), according to a long-term study. The research was published in an article, “…

Treatment of Pseudomonas aeruginosa lung infections in cystic fibrosis (CF) patients could be more effective if done within a crucial two- to three-year period when the bacteria are still susceptible to antibiotics, according to a study. The study, “Evolutionary highways to persistent bacterial infection,” was published in…

Using lower, or suboptimal, doses of antibiotics to treat lung infections in children and young adults with cystic fibrosis (CF) leads to fewer changes in airway bacterial diversity compared to therapeutic (higher) antibiotic exposure. The findings also suggested, however, that patients receiving therapeutic doses had greater improvements in lung…

Stimulation of neurons accounts for approximately half of the beneficial production of airway surface liquid (ASL) induced by hypertonic saline (HTS), a therapeutic agent used for cystic fibrosis (CF) treatment, according to research. The study, “Nebulized hypertonic saline triggers nervous system-mediated active liquid secretion in cystic fibrosis swine…

An exploratory Phase 1 trial showed that repeated intranasal treatment with the therapy eluforsen improved CFTR protein activity in adults with cystic fibrosis (CF) carrying F508del mutations in both CFTR gene copies. The study, “Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis,” was…