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…
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Note: This story has been updated Aug. 11, 2023, to correct the definition of a rare disease, which is defined as one affecting fewer than 200,000 people in the U.S., and to reflect that researchers estimate cystic fibrosis may affect about 160,000 people worldwide. Of the more than 100 investigational…
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…
A year of treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor) led to gains in lung function and nutritional status for cystic fibrosis (CF) patients in Germany, a real-world study in over 2,600 people reported. Sold as Trikafta in the U.S. and Canada, Kaftrio’s use also resulted in fewer in pulmonary exacerbations…
CFTR modulator therapies can enhance the ability of certain immune cells to kill bacteria, independent of defects in the CFTR protein that cause cystic fibrosis (CF), according to a new study. Specifically, these modulators alter metabolic processes to activate anti-bacterial processes within both CF and non-CF immune cells known…
The annual number of new cases of nontuberculous mycobacteria (NTM) lung infections is increasing in the U.S. among people with cystic fibrosis, a study finds. Between 2010 and 2019, the incidence, or new cases, of infections increased by 3.5% a year, with the highest number seen in the South. NTN…
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…
Two new Strategic Research Centres (SRC), each leading a multiyear study into research priorities identified by cystic fibrosis (CF) patients, will open the U.K. in September. The work, which bring together scientists and specialists from different fields, is supported by £1.4 million — around $1.8 million — in funding…
An increase in body mass index (BMI) in people with cystic fibrosis (CF) under Kaftrio therapy may not be attributable to increased energy intake, according to a new U.K. study. Instead, gains in BMI, a measure of body fat, might reflect other factors, such as different energy requirements…
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