Marisa Wexler, MS,  senior science writer—

A novel treatment strategy for cystic fibrosis (CF) that’s based on a cellular structure called the porosome showed promise in cell and animal models of CF caused by the common F508del mutation. In the cell model, this approach was more potent than tezacaftor and ivacaftor, which are CFTR…

Biopharma and biotech companies have launched more than 450 clinical trials into potential new treatments for cystic fibrosis (CF) since 2018, according to a report from Novotech, a contract research organization that specializes in helping drug developers to run trials. “There are ample opportunities presented by advancements in…

The CFTR modulator therapy Kalydeco (ivacaftor) can safely be given to babies with cystic fibrosis (CF) as young as 4 weeks old, according to data from a Phase 3 clinical trial. The findings were the basis of the U.S. Food and Drug Administration (FDA)’s decision to expand…

While Kaftrio — sold as Trikafta in North America — can improve lung function in children with cystic fibrosis (CF), treatment effects are unlikely to be as dramatic in real-world practice as those reported in clinical trials. Rather, for eligible children without substantial lung problems, Kaftrio’s use can…

Mutations that cause cystic fibrosis (CF) lead to biochemical changes in lung cells that make it easier for infectious bacteria to stick to the cells, a new study reports. Findings notably suggest that treatment with Trikafta (ivacaftor/tezacaftor/elexacaftor) did not reverse these changes, which may help to explain why…

The U.S. Food and Drug Administration (FDA) has given the go-ahead for a Phase 2 trial testing the inhalation therapy SPL84 in people with cystic fibrosis (CF) caused by a specific mutation called 3849+10 kb C-to-T. Therapy developer Splisense welcomed the FDA’s clearance of the clinical trial, and…

For adults with cystic fibrosis (CF), the symptoms with the biggest impact on quality of life include pain, fatigue, shortness of breath, and mental health challenges, leading researchers to recommend routine screening to assist CF patients who could benefit from support. “Screening for prevalent symptoms that affect quality of life…

Bacteria in the airways of people with cystic fibrosis (CF) can interfere with the growth of Pseudomonas aeruginosa by secreting acetic acid, a main ingredient in vinegar. That’s according to “Airway commensal bacteria in cystic fibrosis inhibit the growth of P. aeruginosa via a released metabolite,”…

4D Molecular Therapeutics (4DMT) is planning a Phase 3 clinical trial that, if results are positive, will be the basis of applications seeking approval of the company’s inhaled gene therapy 4D-710 as a treatment for people with cystic fibrosis (CF) who can’t take CFTR modulators. 4DMT…

Sionna Therapeutics has raised $182 million in financing to advance the development of small molecules designed to restore CFTR protein function by binding to a specific part of the protein called the first nucleotide-binding domain, or NBD1. Cystic fibrosis (CF) is caused by mutations in the gene…