Andrea Lobo, PhD,  science writer—

The first patient has been dosed in a Phase 1 clinical trial of KB407, Krystal Biotech’s investigational gene therapy for people with cystic fibrosis (CF) regardless of the type of disease-causing CFTR gene mutation. The Phase 1 CORAL-1/US study (NCT05504837) is recruiting up to 20 adults with…

The presence of intestinal metabolites called bile acids in the airways of 1-year-old children genetically diagnosed with cystic fibrosis (CF) is significantly associated with early disease mechanisms and worse clinical outcomes, according to a study in Australia. These include increased levels of airway inflammation biomarkers, greater structural lung damage,…

Kaftrio (elexacaftor, tezacaftor, and ivacaftor) safely and effectively eases structural lung damage and improves lung function and the quality of life for people with cystic fibrosis (CF) who have advanced lung disease. That’s according to data from a compassionate use program in the Netherlands that let patients with severely…

Antibiotic resistance is high among cystic fibrosis (CF) patients infected with Staphylococcus aureus (S. aureus), according to a recent study. There was “high resistance to most of the antibiotics studied,” researchers say, with the highest observed for the antibiotics erythromycin and clindamycin, while the lowest resistance was seen with…

Reducing the dose of Kaftrio (elexacaftor, tezacaftor, and ivacaftor) should be considered for cystic fibrosis (CF) patients who report cognitive and psychological side effects, a study in Ireland suggests. Such patients should have an individualized dosage to minimize side effects while maintaining CFTR modulator therapy. “Dose reduction…

The Cystic Fibrosis Foundation is funding up to $3.9 million to TB Alliance to conduct preclinical studies of a compound that could be used to treat infections caused by nontuberculous mycobacteria (NTM) in people with cystic fibrosis (CF). These infections often require antibiotic treatment for more…

First Wave BioPharma has completed patient screening for the Phase 2 SPAN clinical trial of a new formulation of adrulipase to treat exocrine pancreatic insufficiency (EPI) in cystic fibrosis (CF) patients. The trial (NCT05719311) will be conducted at three sites in Florida, Illinois, and Nevada, and include…

The U.S. Food and Drug Administration (FDA) has approved the expanded use of Trikafta (elexacaftor, tezacaftor, and ivacaftor) to children with cystic fibrosis (CF) ages 2 to 5, who have at least one F508del mutation in the CFTR gene or a mutation that responds to Trikafta in laboratory studies.

SPRIM Global Investments (SGI) has committed $4.5 million to initiate Spexis’ Phase 3 COPILOT clinical trial to test the antibiotic ColiFin in people with cystic fibrosis (CF) who have chronic Pseudomonas aeruginosa lung infections. The trial will test once or twice daily dosing of ColiFin (inhaled colistimethate sodium),…

The Cystic Fibrosis Foundation (CFF) is investing up to $15.5 million in Anagram Therapeutics to conduct early-stage trials of a new oral enzyme replacement therapy. The company is planning to start a Phase 1 clinical trial this summer. The therapy would let people with cystic fibrosis (CF)…