Marisa Wexler, MS,  senior science writer—

A novel compound that may hold promise for treating the roughly 11% of cases of cystic fibrosis (CF) that are caused by nonsense mutations has been described in a new study. The study, “A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1…

Eloxx Pharmaceuticals announced it expects to share data later this year from the first four treatment groups of a Phase 2 program evaluating the investigational therapy ELX-02 in people with cystic fibrosis (CF) who have at least one G542X mutation. “Given the substantial efforts of our clinical team,…

Health Canada has approved Trikafta  as a triple-combination therapy for people with cystic fibrosis (CF), ages 12 and up, who have at least one F508del mutation, the most common CF-causing mutation. This decision is expected to give about 1,100 people access for a first time to a therapy that directly…

A new strategy to correct so-called splicing mutations could help deliver nucleic acid therapies called oligonucleotides to cells in the lung, a new study suggests. This approach may aid in the development of new treatments for people with cystic fibrosis (CF), according to researchers. “With our oligonucleotide delivery platform,…

A collaboration between the Cystic Fibrosis Foundation and Deep Science Ventures aims to identify technologies that might overcome the challenges inherent in developing gene therapies for cystic fibrosis (CF). The effort is part of the foundation’s Path to a Cure initiative, a $500 million research program whose focus…

BX004, a phage therapy candidate for Pseudomonas aeruginosa infections in people with cystic fibrosis (CF), was able to effectively kill a range of antibiotic-resistant bacterial strains under laboratory conditions, new data show. These findings were shared in the poster “Phage therapy for chronic Pseudomonas aeruginosa infections in Cystic Fibrosis…

The first participants have been dosed in the first clinical trial testing EDT001, an investigational therapy for cystic fibrosis (CF) being developed by Enterprise Therapeutics. The trial is being conducted in healthy participants (without CF) to assess the investigational medicine’s safety profile. “We are excited to be entering…

The U.S. Food and Drug Administration (FDA) has approved expanding the use of Trikafta (elexacaftor, tezacaftor, and ivacaftor) to children with cystic fibrosis (CF), ages 6 and older, who have at least one F508del mutation in the CFTR gene or a CFTR mutation that responds to Trikafta in laboratory studies. Use of…

A combination of bacteria-infecting viruses and antibiotics can be used to effectively protect lung cells against infection by Pseudomonas aeruginosa, a bacteria that commonly causes lung infections in people with cystic fibrosis (CF), according to a study led by researchers at the University of Geneva (UNIGE). The model…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…