News

Sionna Therapeutics’ second-generation molecules, combined with standard CFTR modulators, fully restored the production and function of the CFTR protein with the most common mutation that causes cystic fibrosis (CF), according to preclinical data. The Series 2 molecules, SION-719 and SION-451, are undergoing investigational new drug (IND) enabling…

Treatment with the inhaled gene therapy 4D-710 led to clinically meaningful quality of life gains for people with cystic fibrosis (CF) in a clinical trial, according to data announced by 4D Molecular Therapeutics (4DMT), the therapy’s developer. Treatment also led to high expression of the CFTR protein…

The first person with cystic fibrosis (CF) has been dosed with CM001, an experimental inhaled therapy being developed by Cystetic Medicines to treat all people with CF, including those who aren’t eligible for modulators. “Dosing the first person with CF represents a critical milestone in our mission to…

One month of treatment with Trikafta, a triple combo of elexacaftor, tezacaftor, and ivacaftor, helped to clear mucus from the airways of adolescents and adults with cystic fibrosis (CF), a study found. Mucus became less thick, which may have helped in preventing its buildup in the airways, increasing…

Six months of treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) significantly improved lung function and nutritional status in two adults with cystic fibrosis (CF) caused by the rare mutation M1101K, according to two cases described in Canada. “Our cases not only demonstrate the therapeutic benefit of [Trikafta] in individuals with non-F508del…

Trikafta, a combination of elexacaftor, tezacaftor, and ivacaftor, lowered the number of Pseudomonas aeruginosa bacteria in the nose and sinuses of people with cystic fibrosis (CF) taking part in a small study. Mucus from the sinuses — air-filled pockets in the skull — can enter the lungs, so…

The pediatric and adult cystic fibrosis programs at Geisinger Medical Center have received an award from the Cystic Fibrosis Foundation (CFF) for their work in improving the care of people with cystic fibrosis (CF). Given annually, the CFF’s Quality Improvement Award recognizes centers that work continuously to improve…

A high degree of genetic diversity was found among strains of Pseudomonas aeruginosa bacteria initially infecting the lungs of people with cystic fibrosis (CF), a study reports. Over a seven-year follow-up, the majority of CF patients experience a recurrence of the bacteria, with one-third of those being infected…

Moligo Technologies is teaming up with Nationwide Children’s Hospital on a new project to develop a non-viral gene therapy approach for treating cystic fibrosis (CF) and other diseases that affect the lungs. Instead of using a modified virus to deliver a gene therapy into a patient’s body, the…

Parents of adolescents and young adults with cystic fibrosis (CF) retain a sense of responsibility for their children as they transition into adult healthcare services, and they can be frustrated by the changes demanded, an interview study reported. Healthcare providers should recognize the evolving roles of both patients and…