News

Kaftrio seen to ease bronchiectasis on sensitive MRI scans

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…

NACFC 2023: Blocking ferroptosis may treat bacterial infections

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,…

NACFC 2023: Sionna’s Series 2 molecules can restore CFTR protein

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…

First CF patient dosed with molecular prosthetic CM001

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…