Patricia Inácio, PhD,  science writer—

Vertex Pharmaceuticals is launching a clinical trial to test VX-522, its inhaled messenger RNA (mRNA) therapy for lung disease in cystic fibrosis (CF) patients who are not eligible for treatment with an existing CFTR modulator. The study is expected to open soon, and it will assess VX-522’s safety and…

Twice-weekly sessions of airway clearance physiotherapy over eight weeks led to clinically meaningful reductions in respiratory symptoms in people with cystic fibrosis (CF) in a small clinical trial. These preliminary findings, based on patient-reported measures, support the need for further studies to determine the most effective airway clearance technique,…

Prolonged use of certain medications used to treat acid reflux, called proton pump inhibitors (PPIs), may put people with cystic fibrosis (CF) at higher risk for Pseudomonas aeruginosa infection, according to a single-center study in Italy. Long-term treatment was also associated with more hospitalizations for pulmonary exacerbations, or episodes of…

Carmine Therapeutics has completed series A financing to support the development of its innovative, non-viral gene therapy for cystic fibrosis (CF) and other diseases. The company is working to advance a novel type of disease-modifying gene therapy, which is accomplished using its proprietary Red Cell EV Gene Therapy…

First Wave BioPharma has developed a new optimized formulation of adrulipase, a yeast-derived enzyme designed to treat exocrine pancreatic insufficiency in people with cystic fibrosis (CF). The new formulation ensures that the enzyme is packed in acid-resistant micro-granules and protected from the acid environment of the stomach. The enzyme then reaches…

A compound called beta-sitosterol, abundant in plants, eased the burden of Pseudomonas aeruginosa infection — a common bacteria infecting the lungs of people with cystic fibrosis (CF) — and lessened chronic inflammation in a mouse model. Findings suggest that beta-sitosterol, or BSS, “has the potential to undergo pharmaceutical development…

In children with cystic fibrosis (CF), elevated levels of programmed death protein 1 (PD-1) in cells called macrophages are linked with lung disease, inflammation and infection, a study reports. Blocking PD-1 activation resulted in more efficient killing of Pseudomonas aeruginosa, the most common bacteria found in CF patients.

About 10% of adolescents and young adults with cystic fibrosis (CF) have abnormal eating patterns, according to a single-center study. Results suggested that nutrition-focused subscales of the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a tool commonly used for assessing health-related quality of life in CF, may help identify young patients at risk…

A six-week treatment with Kaftrio led to marked improvement in exercise capacity in adolescents with cystic fibrosis (CF), according to an analysis of three cases. The study, “Elexacaftor–Tezacaftor–Ivacaftor improves exercise capacity in adolescents with cystic fibrosis,” was published in the journal Pediatric Pulmology. CF is caused by mutations in…

Most adults with cystic fibrosis (CF) report experiences with painful or frightening medical procedures in childhood after their diagnosis, according to a U.K. study. These patients may benefit from psychosocial support from CF teams, who can ask about trauma in childhood in a safe and sensitive manner, researchers say.