News

CF Foundation invests $3M in experimental therapy RCT2100

The Cystic Fibrosis Foundation has made an additional $3 million investment in Recode Therapeutics to support clinical development of RCT2100, an investigational therapy for cystic fibrosis (CF) that is currently being tested in patients who cannot take CFTR modulators. The CF Foundation had previously invested $15 million…

Trikafta benefits seen regardless of prior CFTR modulator use for CF

One-year treatment with Trikafta — a widely used, approved medication for people with cystic fibrosis (CF) — significantly eases symptoms and improves outcomes, regardless of whether or not patients were previously treated with other CFTR modulators, a new real-world study reports. Among other benefits, Trikafta — a combination…

Blood test in pregnancy accurately diagnoses CF: Study

The Unity Fetal Risk Screen, a test that uses a pregnant person’s blood to evaluate the fetus’s risk of cystic fibrosis (CF), accurately detected all cases of CF in a study involving more than 100,000 pregnant people. The study found that most CF cases identified by the test are…

Nanoparticles in gene-editing therapy may correct CF defects

Researchers have developed nanoparticles that can deliver gene-editing therapeutics to correct genetic defects in the lungs of people with cystic fibrosis (CF). The nanoparticles were optimized using cell-based models to penetrate the thick airway mucus seen in CF. Pretreating patients with the approved mucus-clearing agent Pulmozyme (dornase alfa)…

SPL84 shows signs of improving lung function in CF patients

SPL84, an investigational inhalation therapy designed to treat cystic fibrosis (CF) caused by a specific mutation called 3849+10 kb C-to-T, was well tolerated in a Phase 2 clinical trial. Trial data also suggest that most CF patients treated with SPL84 experienced improvements in a measure of lung function, according…