Forest Ray PhD,  —

A bioengineer at Rice University will use a more than $2 million federal grant for a project to “repair” harmful mutations that cause cystic fibrosis (CF) using a potentially more accurate approach to gene editing developed in her lab, the university announced in a press release. The National…

Due to persistent bacterial infections, long-chain fatty acids (LCFA) are linked to continued inflammation in cystic fibrosis (CF) patients — and may contribute to pulmonary exacerbations in children with CF, according to a recent study. The scientists said further studies are needed to examine how bacterial functions following antibiotic…

AzurRx BioPharma announced that its Phase 2b trial evaluating the safety and efficacy of MS1819 in treating exocrine pancreatic insufficiency (EPI) in cystic fibrosis (CF) patients is fully enrolled. Thirty adults with CF are taking part in the OPTION 2 study (NCT04375878) comparing MS1819 with the current standard…

A recent survey shows that people with cystic fibrosis (CF) broadly favor targeted therapy using patient-derived organoids — the three-dimensional organ-like structures made from a patient’s own cells. The finding suggests that this technology might be integrated successfully into healthcare systems. The study, “Avatar acceptability: views…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Individuals with cystic fibrosis (CF) from racial and ethnic minority groups in the U.S. are twice as likely to be ineligible for disease-modifying therapies than white patients, because their mutations are not known to be suited to approved treatments, a study reports. The resulting lack of treatment access exacerbates…

Researchers boosted the activity of the cystic fibrosis transmembrane regulator (CFTR), the faulty protein at the heart of cystic fibrosis (CF), by altering the levels of a molecule called cyclic AMP (cAMP). Their results suggest that cAMP might make an effective add-on therapy to approved CFTR modulators like…

The Cystic Fibrosis Foundation (CFF) has awarded $2.76 million to fund 11 laboratory studies exploring the effects of COVID-19 on people with cystic fibrosis (CF). The hope is that a better understanding of why differences in COVID-19 severity exist among people with CF will ultimately lead to…

Experimental therapy icenticaftor (QBW251) was able to improve lung function in cystic fibrosis (CF) patients carrying class 3 and 4 mutations in the CFTR gene, results from a Phase 1/2 trial show. It also appeared to be safe and well tolerated when given to both healthy volunteers and CF…

Vertex Pharmaceuticals has awarded a $50,000 grant to Claire’s Place Foundation to support its Work Proudly Program, which helps adults with cystic fibrosis (CF) and caregivers with work-from-home opportunities. “We are extremely grateful for the influx of support for our new Work Proudly Program,” Melissa Yeager,…