Kaftrio, a CFTR modulator marketed as Trikafta (elexacaftor/tezacaftor/ivacaftor) in the U.S., was associated with improved lung function and nutritional status in a study. Researchers found only small changes in which microorganisms were present, but larger changes in their activity. Those larger changes included more microbial activity related to butyrate,…

The genetic defect that causes cystic fibrosis (CF) may also directly contribute to the development of pancreatic cancer, a new study done in cell models suggests. Researchers found that pancreatic cancer cells carrying CF-causing mutations grow faster than cancer cells without these mutations, and this growth could be…

GLP-1 receptor agonists (GLP-1RAs), medications commonly used for diabetes and weight loss, may improve lung function in adults with cystic fibrosis-related diabetes (CFRD), according to a small, exploratory study from Australia. After one year of treatment, patients given a GLP-1RA in addition to insulin, the standard CFRD treatment, had significant…

The next-generation triple-combination CFTR modulator Alyftrek can be safely given to children with cystic fibrosis (CF) as young as 2, according to results from a Phase 3 clinical trial. Findings from the trial also suggest that Alyftrek can improve CFTR activity in kids who switch from the first-generation…

The Cystic Fibrosis Foundation has awarded University of Michigan researchers $900,000 over two years to advance new treatments for cystic fibrosis (CF) — including genetic therapies that could offer novel options for people who do not benefit from existing medicines. “There are many different mutations that cause CF and several types of…

CFTR modulators for cystic fibrosis (CF) can change a child’s weight and nutrition needs, so each child should receive an individualized nutrition plan based on how they are growing, what they prefer to eat, and how they are responding to treatment, a survey study found. The study, “Parents’/Caregivers’…

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…

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…