Patricia Inácio, PhD,  science writer—

The Cystic Fibrosis Foundation (CFF) has awarded funding to enGene to support its work on a non-viral and inhalable gene therapy that could deliver a working copy of the CFTR gene to the lungs of people with cystic fibrosis (CF). “Today’s announcement reflects yet another milestone on our path…

A Phase 2 clinical trial investigating MS1819 in combination with stable pancreatic enzyme replacement therapy (PERT) to treat exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF) is actively recruiting participants in Europe, AzurRx BioPharma, the treatment’s developer, announced. The open-label study (NCT04302662) expects to enroll 24 CF patients, ages 12…

Factors such as diabetes, a specific type of fungal infection, and previous mild to moderate episodes of hemoptysis, or coughing up blood, may help predict the risk of massive hemoptysis in people with cystic fibrosis (CF), a study suggests. The study, “Predictors of massive haemoptysis after…

ContraFect will receive funding from the Cystic Fibrosis Foundation to conduct preclinical research about the potential of direct lytic agents, an alternative to conventional antibiotics, to treat lung infections caused by Gram negative antibiotic-resistant bacteria, including Pseudomonas aeruginosa. While antibiotics have had a profound impact on…

New research unveils why the bacteria Pseudomonas aeruginosa cause sustained infections in younger cystic fibrosis (CF) patients, while the bacteria Burkholderia cepacia complex infects patients as teenagers and adults. The findings show that P. aeruginosa loses the ability to use its toxic weaponry after it adapts to the lungs, allowing Burkholderia cepacia complex to…

A new study will assess the real-world effects of Vertex Pharmaceutical‘s Trikafta, a triple combo of elexacaftor, tezacaftor, and ivacaftor approved by the U.S. Food and Drug Administration (FDA) for treating up to 90% of all people with cystic fibrosis (CF). The $3.63 million study (3.06…

The Welsh government and Vertex Pharmaceuticals have reached an agreement that will allow access to the company’s Kaftrio (ivacaftor/tezacaftor/elexacaftor) for cystic fibrosis (CF) patients as soon as the medication is approved in Europe. Kaftrio is a triple combination therapy for the treatment of CF, designed to…

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…