The Cystic Fibrosis Foundation is investing up to $7.3 million in Ride Therapeutics to develop nonviral carriers of genetic therapies intended to reach the lungs for all people with cystic fibrosis (CF). Ride said its technology could ultimately make genetic medicines more feasible in CF by improving the delivery of therapeutic…
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Most adults with cystic fibrosis (CF) surveyed at a U.S. care center had never received genetic counseling, and many had unanswered questions about fertility, pregnancy, and family planning. Among more than 100 respondents, 71% had not received genetic counseling, and 27% had never heard of it. Fifty-four percent of…
Two drug developers are teaming up to advance toward clinical trials an inhaled treatment targeting the disease mechanisms underlying cystic fibrosis (CF). The U.S.-based Aptar Pharma and Aceso Therapeutics, headquartered in France, announced they will collaborate on developing ACT-101, an experimental RNA therapy. Unlike traditional drugs that target…
Children and adolescents with cystic fibrosis (CF) continue to experience mental health and behavioral concerns despite treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor), according to a recent study. Caregiver anxiety and depression were also found to be associated with mental health and behavioral symptoms in their children, highlighting the importance of…
Higher than normal levels of several pH-regulating proteins can be found in the bronchial, or airway, cells of people with cystic fibrosis (CF), according to a new study. This suggests the body may be compensating for the loss of CFTR function — which helps regulate the movement of salt…
An experimental RNA-based approach restored production and function of CFTR — the protein that is faulty or missing in cystic fibrosis (CF) — in preclinical models carrying disease-causing nonsense mutations, a study shows. These mutations introduce an incorrect stop signal in the genetic instructions for making CFTR, causing cells…
Bacterial extracellular vesicles (bEVs) — tiny sacs released by bacteria containing pro-inflammatory molecules — have been found at elevated levels in the bloodstream of people with cystic fibrosis (CF), particularly in those whose lungs are infected with the bacterium Pseudomonas aeruginosa. In the pilot study, the researchers call…
A single dose of an inhaled experimental gene therapy for cystic fibrosis (CF) — one designed to treat people regardless of their disease-causing genetic mutation — was shown in an animal model to sustainably boost CFTR gene activity and protein levels in the lungs and spread to several other…
Two U.S.-based researchers aiming to help gene-corrected airway stem cells grow and survive long enough to repair lung tissue — with the goal of developing a long-lasting treatment for cystic fibrosis (CF) — have received a grant from the nonprofit Emily’s Entourage. This type of stem cell therapy may…
A study found machine learning models could help predict whether chronic lung infections in people with cystic fibrosis (CF) will be resistant to commonly used antibiotics, helping doctors make more informed treatment choices. By linking a decade of information on patients’ antibiotic resistance with previous antibiotic use and other…
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