News

ViaNautis has secured $25 million in financing to advance work with polyNaut, its delivery platform for genetic and other treatments. The platform is designed to deliver a range of therapies into difficult-to-penetrate tissues. According to the company, the money raised will help to support polyNaut’s use…

A newborn screening program in New Jersey failed to identify cystic fibrosis (CF) in five children, all of whom went on to have severe lung disease, a new study reports. Based on the findings, New Jersey’s newborn screening program has been updated to include screening for many more disease-causing…

An MRI technique revealed signs of reversible bronchiectasis (widened and inflamed airways) in more than one in three people with cystic fibrosis (CF) being treated with Kaftrio, sold as Trikafta in the U.S., as part of a small one-year study in France. The technique, called ultrashort echo-time MRI, is…

Six months of treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor), sold as Trikafta in the U.S., leads to increases in body mass index (BMI) and fat mass in people with cystic fibrosis (CF), a small study has found. However, while BMI increased for both men and women, only women showed a…

Researchers hope a new tool that can provide disease-specific education and resources about a lung transplant for people with cystic fibrosis (CF) will be able to make inroads among patients who’ve historically had poor access to the procedure. The development of the tool, called Take on Transplant, was introduced…

Blocking iron-dependent cell death, a process called ferroptosis, may treat antibiotic-resistant Pseudomonas aeruginosa lung infections in people with cystic fibrosis (CF). “Blocking ferroptosis might be a really promising and unique new way to treat [Pseudomonas aeruginosa] infections,” said Peter A. Jorth, PhD, from Cedars-Sinai Medical Center, California,…

Long-term Trikafta treatment led to sustained improvements in CFTR function and reduced lung symptoms in children, ages 6-11, with cystic fibrosis (CF), according to almost three years of interim data from an open-label extension (OLE) study. There were no signs of lung function decline during the 3.5-year treatment…

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…