Patricia Inácio, PhD,  science writer—

The first two patients have been dosed in the SPAN clinical trial testing a new formulation of adrulipase, a yeast-derived enzyme, to treat exocrine pancreatic insufficiency (EPI) in adults with cystic fibrosis (CF). The open-label Phase 2 study (NCT05719311) is enrolling adult patients at three sites, one each in Florida, Illinois,…

AP-PA02, an experimental phage therapy for people with cystic fibrosis (CF) and chronic Pseudomonas aeruginosa respiratory infections, was well tolerated and reduced the bacterial load in the lungs, according to top-line results from a Phase 1b/2a trial. High doses of AP-PA02 were associated with durable reductions in bacterial load in the SWARM-Pa study. Pharmacokinetics data,…

CDX-7108, an oral therapy being developed by Codexis for exocrine pancreatic insufficiency (EPI) — which is common in people with cystic fibrosis (CF) — may safely improve fat absorption, according to interim data from an ongoing Phase 1 clinical trial. The trial, sponsored by Nestlé Health Science,…

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…