Author Archives: Forest Ray PhD

Arrowhead Pharmaceuticals has voluntarily paused new screening, enrollment, and dosing in its Phase 1/2 trial of ARO-ENaC, an investigational treatment for cystic fibrosis (CF). The decision came after unexpected signs of local lung inflammation occurred in an ongoing chronic toxicology study taking place in rats. “The safety of…

Treatment with Orkambi (ivacaftor/lumacaftor) led to significant improvements in lung health, particularly related to clearing mucus, among people with cystic fibrosis (CF), according to a real-world study of its use in France. The investigation provided a first high-resolution CT scan-based assessment of the medication’s effectiveness, and results also indicated…

Physical activity, undertaken regularly over the long term, appears to complement other therapies in managing cystic fibrosis (CF), a study in a group of active and non-active patients found. Its results also showed that tumor necrosis factor alpha (TNF-alpha) may serve as a biomarker of inflammation to monitor the…

Regulatory agencies in the European Union and in the U.K agreed to consider expanding the use of Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with Kalydeco (ivacaftor), to children with cystic fibrosis (CF) who have at least one F508del mutation starting at age 6. The European Medicines Agency (EMA) and the U.K.’s Medicines…

People living with cystic fibrosis (CF) are invited to participate in CF Awareness Month by submitting entries for the CF Awareness Film Festival, taking place virtually May 24–31. Supported by the Bonnell Foundation and the I See You Awards, the festival aims to highlight issues faced by…

People living with cystic fibrosis (CF) need clear, aggressive, and science-driven COVID-19 vaccine distribution strategies that prioritize their condition, says a coalition of experts. The Global Registry Harmonization Group (GRHG), composed of scientists and patient advocates, including the CF Trust, wrote a letter to the…

A bioengineer at Rice University will use a more than $2 million federal grant for a project to “repair” harmful mutations that cause cystic fibrosis (CF) using a potentially more accurate approach to gene editing developed in her lab, the university announced in a press release. The National…

Due to persistent bacterial infections, long-chain fatty acids (LCFA) are linked to continued inflammation in cystic fibrosis (CF) patients — and may contribute to pulmonary exacerbations in children with CF, according to a recent study. The scientists said further studies are needed to examine how bacterial functions following antibiotic…

AzurRx BioPharma announced that its Phase 2b trial evaluating the safety and efficacy of MS1819 in treating exocrine pancreatic insufficiency (EPI) in cystic fibrosis (CF) patients is fully enrolled. Thirty adults with CF are taking part in the OPTION 2 study (NCT04375878) comparing MS1819 with the current standard…

A recent survey shows that people with cystic fibrosis (CF) broadly favor targeted therapy using patient-derived organoids — the three-dimensional organ-like structures made from a patient’s own cells. The finding suggests that this technology might be integrated successfully into healthcare systems. The study, “Avatar acceptability: views…