The U.S. Food and Drug Administration (FDA) has expanded the number of cystic fibrosis (CF)-related mutations considered responsive to and therefore eligible for Vertex Pharmaceuticals‘ three approved therapies. With these approvals, the labels will be expanded for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor), and…
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Scientists discovered the subtypes of gentamicin — a broad-spectrum antibiotic often used to treat bacterial infections in people with cystic fibrosis (CF) — that are more likely to cause deafness, and created a new formulation that is as effective at eliminating bacteria but much safer for patients. “We’ve developed…
The U.K. Cystic Fibrosis Trust is partnering with the U.S. Cystic Fibrosis Foundation to fund a Strategic Research Centre (SRC) — a virtual collaboration bringing together investigators with different backgrounds and areas of expertise — to advance the development of gene-editing therapies for treating cystic fibrosis…
Project GAIA, establishing a large-scale manufacturing process for Pyocin S5, an investigational antibiotic for the treatment of lung disease in people who have cystic fibrosis, is now complete. The scale-up process for Pyocin S5 manufacturing was performed by CPI, an independent technology innovation center in the U.K. This work will…
A newly developed antibiotic, called T405, appears to effectively kill Mycobacteroides abscessus, a difficult-to-treat bacteria that can cause serious infections in people with lung conditions like cystic fibrosis (CF). The antibiotic was described in the study “Development of a penem antibiotic against Mycobacteroides abscessus,” published in Communications…
The Cystic Fibrosis Foundation has awarded a total of $1.7 million in nine grants to research projects focused on tools and strategies to advance therapies for all cystic fibrosis (CF) patients, regardless of their underlying mutations. “There has been an explosion of scientific progress in novel technologies with…
The Cystic Fibrosis Foundation (CFF) has awarded $2.76 million to fund 11 laboratory studies exploring the effects of COVID-19 on people with cystic fibrosis (CF). The hope is that a better understanding of why differences in COVID-19 severity exist among people with CF will ultimately lead to…
Specific immune cells called macrophages in cystic fibrosis (CF) are unable to kill the bacteria Burkholderia cenocepacia due to impairments in mitochondria, the cell’s powerhouses, a new mouse study says. The finding suggests that boosting mitochondria function could help resolve chronic infection in CF. The study “Defective immunometabolism…
Children with cystic fibrosis (CF) who become infected with SARS-CoV-2 — the virus that causes COVID-19 — usually have a mild disease course, a new study reports. The findings also indicate that the risk of severe disease may be highest in children with CF who have poorer lung function…
Experimental therapy icenticaftor (QBW251) was able to improve lung function in cystic fibrosis (CF) patients carrying class 3 and 4 mutations in the CFTR gene, results from a Phase 1/2 trial show. It also appeared to be safe and well tolerated when given to both healthy volunteers and CF…
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