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A lung-targeted gene-editing therapy designed using technology from ReCode Therapeutics was able to successfully correct a disease-causing CFTR gene mutation in lung cells derived from cystic fibrosis (CF) patients, according to a recent proof-of-concept study. The therapy, developed using ReCode’s Selective Organ Targeting (SORT) lipid nanoparticles (LNPs), was…

Low doses of ETD001, an inhaled medication being develop by Enterprise Therapeutics to treat cystic fibrosis (CF), acted for longer than expected and helped to clear mucus from the airways of sheep in a study. Equivalent doses also were well tolerated in an earlier Phase…

During pulmonary exacerbation in cystic fibrosis (CF), bacterial communities in the lungs tend to change in one of two general patterns, and better understanding these patterns may help predict how patients will respond to antibiotic treatments. That’s according to the study, “Microbial community organization designates distinct…

Scientists at the Broad Institute of MIT and Harvard have created a gene editing technology that may be able to insert or substitute entire genes in human cells, which could pave the way for new gene therapies for diseases such as cystic fibrosis (CF). The approach could efficiently insert…

ARCT-032, a messenger RNA inhalation treatment being developed by Arcturus Therapeutics to restore a working protein in the lungs of cystic fibrosis (CF) patients, showed safety in healthy adults and early signs of potential efficacy in a small group of patients taking part in a Phase 1/1b…

CF AMR Syndicate, a U.K. research support group, has awarded BioVersys up to £500,000 ($636,000) to develop small molecules targeting hard-to-treat nontuberculous mycobacteria (NTM) infections in people with cystic fibrosis (CF). Established in 2019 as a cross-sector (e.g., public-private) initiative with the Cystic Fibrosis Trust to…

A single dose of 4D Molecular Therapeutics’ inhaled gene therapy candidate 4D-710 improved or stabilized lung function in adults with cystic fibrosis (CF), according to interim data from a Phase 1/2 clinical trial. Additional data from the AEROW (NCT05248230) study, which is evaluating the therapy in CF…

Levels of fat-soluble vitamins D and E and of the micronutrients zinc and selenium are generally within the normal range in people with cystic fibrosis (CF), even during treatment with Kaftrio. That’s according to a recent study that also found significantly increased vitamin A levels one year into…

Extended treatment with Kaftrio progressively increases the diversity of the microorganisms in the gut in people with cystic fibrosis (CF), a recent study suggests. However, gut microbiota diversity remained significantly different from healthy people after about 1.5 years on the CFTR modulator, probably due to the use…

Women with cystic fibrosis (CF) who have been pregnant three or more times are more likely to experience a greater decline in lung function and, when 30 or older, to give birth to preterm babies or babies with complications, a study has found. Lung function appeared to decline more…