Patricia Inácio, PhD,  science writer—

Patient dosing has begun in a Phase 1b trial evaluating Calithera Bioscience’s oral therapy CB-280 for cystic fibrosis (CF) patients with chronic airway infections, the company announced. The study (NCT04279769), currently enrolling eligible adults at its two U.S. sites, will test four escalating…

Orkambi (ivacaftor/lumacaftor) can significantly reduce the number of days people with cystic fibrosis (CF) require intravenous antibiotics for flares and helps with weight gain — independently of a patient’s level of lung function at the start of treatment, a real-world study reported. While the greatest lung improvements were…

Exposure to second-hand smoke in infants and children with cystic fibrosis (CF) is linked to changes in levels of metabolites and activation of signaling pathways that enhance inflammation, a study has found. The findings suggest exposure to second-hand smoke is a potentially important environmental factor in CF. The study…

The antibiotic-resistant bacterium Stenotrophomonas maltophilia, a dangerous bacteria for people with lung diseases such as cystic fibrosis (CF), is widely spread around the world, with most strains carrying virulence and resistance genes, a large genetic study shows. The study, “The phylogenetic landscape and nosocomial spread of…

Arrowhead Pharmaceuticals has filed for approval to launch a Phase 1/2a clinical trial in New Zealand testing its candidate inhaled therapy for cystic fibrosis (CF), called ARO-ENaC, the company announced. ARO-ENaC is an investigational RNA therapy designed to lower the production of the epithelial sodium channel alpha subunit (αENaC) in the…

ReCode Therapeutics has raised $80 million in Series A financing to support the preclinical development of its lead targeted RNA therapy for cystic fibrosis (CF). The funding also will be used to support the development of RNA therapies for primary ciliary dyskinesia (PCD), a hereditary condition that impairs the function…

A new variation of the gene-editing technology CRISPR-Cas9 can correct mutations in the CFTR gene — the genetic cause of cystic fibrosis (CF) — in stem cells from CF patients, a study shows. The new approach has the ability to correct mutations without the need to excise the affected region,…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

Amplifiers, a potential new class of therapies that modify CFTR — the protein whose defects cause cystic fibrosis (CF) — do so by stabilizing the template used for its production so the protein works better in cells and there’s more of it, a study into the mechanisms of amplifiers reported.

Girls with cystic fibrosis (CF) under the age of six have higher levels of a tiny RNA molecule, miR-885-5p, in their blood compared with boys of the same age, which could be linked to the worse clinical outcomes normally seen in women with CF, a study suggests. The study,…