Children with cystic fibrosis (CF) on Kaftrio maintained stable lung function over one year despite reducing their use of hypertonic saline and other airway-clearance treatments, a study shows. The reduction was accompanied by fewer days of antibiotic use. Respiratory symptoms and airway bacteria also remained stable, suggesting that…
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Sionna Therapeutics is planning a proof-of-concept Phase 2a clinical trial to test a combination of two experimental treatments for cystic fibrosis (CF). The trial, dubbed AscenSION CF, will enroll adults who carry two copies of F508del, the most common CF-causing mutation, and who are taking the approved…
Splisense has begun testing its inhaled therapy SPL84 alongside standard treatment in adults with cystic fibrosis (CF) who carry a specific genetic mutation. This second phase of an ongoing Phase 2 clinical trial is evaluating whether SPL84 can offer extra benefits when paired with the approved…
The Cystic Fibrosis Foundation is investing up to $7.3 million in Ride Therapeutics to develop nonviral carriers of genetic therapies intended to reach the lungs for all people with cystic fibrosis (CF). Ride said its technology could ultimately make genetic medicines more feasible in CF by improving the delivery of therapeutic…
Most adults with cystic fibrosis (CF) surveyed at a U.S. care center had never received genetic counseling, and many had unanswered questions about fertility, pregnancy, and family planning. Among more than 100 respondents, 71% had not received genetic counseling, and 27% had never heard of it. Fifty-four percent of…
Two drug developers are teaming up to advance toward clinical trials an inhaled treatment targeting the disease mechanisms underlying cystic fibrosis (CF). The U.S.-based Aptar Pharma and Aceso Therapeutics, headquartered in France, announced they will collaborate on developing ACT-101, an experimental RNA therapy. Unlike traditional drugs that target…
Children and adolescents with cystic fibrosis (CF) continue to experience mental health and behavioral concerns despite treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor), according to a recent study. Caregiver anxiety and depression were also found to be associated with mental health and behavioral symptoms in their children, highlighting the importance of…
Higher than normal levels of several pH-regulating proteins can be found in the bronchial, or airway, cells of people with cystic fibrosis (CF), according to a new study. This suggests the body may be compensating for the loss of CFTR function — which helps regulate the movement of salt…
An experimental RNA-based approach restored production and function of CFTR — the protein that is faulty or missing in cystic fibrosis (CF) — in preclinical models carrying disease-causing nonsense mutations, a study shows. These mutations introduce an incorrect stop signal in the genetic instructions for making CFTR, causing cells…
Bacterial extracellular vesicles (bEVs) — tiny sacs released by bacteria containing pro-inflammatory molecules — have been found at elevated levels in the bloodstream of people with cystic fibrosis (CF), particularly in those whose lungs are infected with the bacterium Pseudomonas aeruginosa. In the pilot study, the researchers call…
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