Marisa Wexler, MS,  senior science writer—

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…

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…

A new study called ENHANCE is seeking to better understand the current effect of cystic fibrosis (CF) in young children. “There is so much to learn about the changes that are happening in children with CF, we are really excited about this new study,” Paul McNally, MD, a professor…

Researchers have developed new synthetic molecules that can transport chloride salts across cell membranes, which showed promise for restoring normal mucus production in a cell model of cystic fibrosis (CF), a new study shows. “One day, [these molecules] could be leveraged into a drug that treats cystic fibrosis,” Bing…

Men with cystic fibrosis (CF) commonly have low testosterone levels, though this is likely underrecognized due to a lack of testing, a new study highlights. The findings underscore the importance of checking testosterone levels in men with CF so that interventions can be discussed if warranted, researchers said in…

The lungs of people with cystic fibrosis (CF) harbor stem cells with a distinctive proinflammatory genetic signature, a new study shows. Data indicate that the proinflammatory genetic profile of these stem cells is not affected by treatment with CFTR modulators, a class of medications that can boost the…

ReCode Therapeutics has raised $50 million in extended series B financing, which the company plans to use to help develop RCT2100, its experimental treatment for cystic fibrosis (CF). “We are delighted with the continued high level of interest in our novel approach to the targeted delivery of genetic…

A type of bacteria called Achromobacter, which can cause problematic lung infections in people with cystic fibrosis (CF), uses a molecular weapon — one known as a type 3 secretion system — to induce a powerful inflammatory response during infections, a new study found. “These bacteria resist the action…

CFTR — the protein whose defect or absence causes cystic fibrosis (CF) — helps cells called ionocytes remove liquid from the airways, a study shows. The finding sheds new light on the biological processes that are disrupted in CF and may have important implications for treating the disease. The…