Marisa Wexler, MS,  senior science writer—

A novel gene-editing tool that allows scientists to “drag-and-drop” sequences of DNA into the genome, without inducing large cuts, could aid in treating genetic diseases like cystic fibrosis (CF), a study reported. The technology, aptly called PASTE (for programmable addition via site-specific targeting elements), “expands the capabilities of genome…

Vertex Pharmaceuticals recently filed an application with the U.S. Food and Drug Administration (FDA) asking that Trikafta, its triple-combination modulator therapy, be approved for children with cystic fibrosis (CF) ages 2 to 5. The company is planning to file similar requests in the European Union and U.K.

A new test that measures the excretion of bicarbonate in urine may be used to assess the effectiveness of treatments for cystic fibrosis (CF), according to a new study. “The goal is for the urine test to be used as a clinical tool to determine both the severity of…

Differences in levels of proteins related to wound healing, cell migration and neurological development may influence whether or not people with cystic fibrosis (CF) experience improved lung function with Kalydeco (ivacaftor), according to a new analysis. The analysis found that “differences in…

Treatment with the triple-combination therapy Trikafta (elexacaftor/tezacaftor/ivacaftor) reduces inflammation and promotes lung tissue repair in cystic fibrosis (CF), new research indicates. Nicola Robinson, MD, from The University of Edinburgh, presented the findings at the 2022 North American Cystic Fibrosis Conference, in the talk “Elexacaftor/Tezacaftor/Ivacaftor treatment promotes inflammation…

People with cystic fibrosis (CF) tend to report improvements in health-related quality of life associated with treatment with Trikafta. Reports of worse mental health were common, however, and were identified as an area of need. That’s according to data shared by Aricca Van Citters, from The Dartmouth Institute for Health Policy & Clinical Practice,…

Later this month, Laurent Pharmaceuticals will meet with the U.S. Food and Drug Administration (FDA) to discuss next steps for LAU-7b, the company’s experimental inflammation-controlling therapy for cystic fibrosis (CF) that showed promise in a recent Phase 2 study. “There was no precedent in cystic…

Treatment with LAU-7b, an oral form of fenretinide being developed by Laurent Pharmaceuticals, may help prevent lung function decline in adults with cystic fibrosis (CF), particularly those with mild disease, according to results from a Phase 2 clinical trial. Larry Lands, MD, PhD, the company’s chief medical…

The improvements in lung function, respiratory symptoms, and nutritional status with Trikafta are stable after nearly three years in adolescents and adults with cystic fibrosis (CF), according to new data from a long-term extension study. Results were discussed at the 2022 North American Cystic Fibrosis Conference (NACFC) in the presentation, “…

Six months of treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) led to significant improvements in lung function and a self-reported easing of respiratory symptoms for children with cystic fibrosis (CF) ages 6 to 11 in an observational study of its real-world use. Felix Ratjen, MD, PhD, division chief of pediatric respiratory…