Marisa Wexler, MS,  senior science writer—

Gene therapy to deliver a modified and better working version of the CFTR protein may be more effective in treating cystic fibrosis (CF) than a gene therapy aiming to deliver higher levels of the protein, a study shows. “Gene therapy and gene editing techniques, whilst still in their infancy,…

Porosome Therapeutics is seeking a U.S. patent that would cover its “radically new and distinctive” technology platform for the treatment of cystic fibrosis (CF) and other health conditions, the company said in a press release. Its platform works by targeting a cellular structure called the porosome. The global…

Taking high-dose vitamin D supplements during acute lung infections may help to improve bone health for people with cystic fibrosis (CF). “These findings suggest that use of high-dose vitamin D supplementation during an APE [acute pulmonary exacerbation] may mitigate the bone loss suffered by people with CF with each…

An international team of scientists has published new guidelines for how best to perform exercise testing in people with cystic fibrosis (CF). “Guidance and standard operating procedures for functional exercise testing in cystic fibrosis” was published in European Respiratory Review. “Our goal is that all people…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to BX004, a virus-based therapy that BiomX is developing to treat Pseudomonas aeruginosa bacterial infections in people with cystic fibrosis (CF). The FDA gives this designation to therapies that are designed to address unmet medical…

Treatment with Trikafta (ivacaftor/tezacaftor/elexacaftor) may help to ease a particular type of lung inflammation found in people with cystic fibrosis (CF), called a type 2 inflammatory response, that’s often associated with asthma and allergies. As asthma is reported to affect 30.8% of CF patients, a research team wondered…

New grants awarded by the Cystic Fibrosis Foundation will help fund 10 programs that support people impacted by cystic fibrosis (CF). The awards are being given through the foundation’s eighth annual Impact Grants, which provide as much as $10,000 for up to two years to people and nonprofit…

Scientists have synthesized a new compound that can boost the activity of the CFTR protein carrying the most common mutation that causes cystic fibrosis (CF). In cell models, the compound was able to further increase the activity of approved CFTR modulators. The compound’s development was described in the…

Treatment with the CFTR modulator Kalydeco (ivacaftor) led to significant weight gain, suggesting better nutritional status, in  babies and toddlers with cystic fibrosis (CF) eligible for this therapy’s use, a study reports. “Young children experienced favorable changes in weight gain and some nutritional biomarkers in the first 12…

Treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) can improve lung function and nutritional status for people with cystic fibrosis (CF) caused by the N1303K mutation, a small real-world study reports. The N1303K mutation is not among the 178 disease-causing mutations in the CFTR gene considered to be responsive to Trikafta and…