Patricia Inácio, PhD, science writer —

The nonprofit Emily’s Entourage has awarded $220,000 to a project to develop a nonviral and inhalable gene therapy for cystic fibrosis (CF). The grant was awarded to Lorraine Martin, PhD, professor at Queen’s University Belfast, U.K. Martin is developing the therapy in collaboration with OmniSpirant Therapeutics. The…

The European Commission (EC) has granted orphan drug designation to KB407, Krystal Biotech’s potential inhaled gene therapy for cystic fibrosis (CF). Orphan drug status is intended to encourage the development of therapies for rare, life-threatening, or chronically debilitating conditions, through a series of benefits, such as 10 years of market exclusivity…

A Phase 2 trial of ACG-701, an investigational oral treatment for pulmonary exacerbations in people with cystic fibrosis (CF), is underway at clinical sites in the U.S. The REPRIEVE trial (NCT05641298), launched in December, is evaluating the safety, pharmacokinetics, and effectiveness of ACG-701, being developed by Aceragen, when added…

Physical activity increases fat-free mass — the lean mass in muscles, organs, bones, ligaments and tendons — in adolescents with cystic fibrosis (CF), according to a recent study. The findings also showed that fat-free mass was significantly lower in those with CF-related liver disease. The study, “…

Treatment with CFTR modulators was safe during pregnancy and breastfeeding in two women with cystic fibrosis (CF), according to a recent report. Despite being advised to stop treatment, both women continued on their CFTR modulator during pregnancy and breastfeeding without any safety concerns. According to the researchers, this…

The levels of fecal calprotectin protein increase during pulmonary exacerbations — times when respiratory symptoms suddenly worsen — then drop within two weeks of systemic antibiotic treatment given children and adolescents with cystic fibrosis (CF), a study from Iran reported. “Fecal calprotectin level could be considered as a…

SpliSense has launched a Phase 1/2 clinical trial to test SPL84, its inhaled treatment candidate for cystic fibrosis (CF) patients carrying a common disease-causing splicing mutation. As of now, no specific treatment is available for people with CF who carry this particular mutation. Called 3849+10 kb C-to-T, it…

The Cystic Fibrosis Foundation has invested another $4.85 million — termed an equity investment — in Aridis Pharmaceuticals to advance the clinical development of AR-501, an inhaled treatment for chronic lung infections in people with cystic fibrosis (CF). The total funding from the CF Foundation for the clinical…

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,…