Marta Figueiredo, PhD, science writer —

SpliSense has raised $28.5 million in funding to advance its antisense oligonucleotide (ASO) platform for the treatment of cystic fibrosis (CF) caused by certain rare mutations, and of other genetic lung diseases. ASOs are molecules designed to target the messenger RNA (mRNA), the intermediate molecule derived from DNA that guides protein production. Money…

Pel, a complex sugar molecule produced by Pseudomonas aeruginosa, contributes to the bacterium’s ability to aggregate and resist both antibiotics and mucus thinners in people with cystic fibrosis (CF) by binding to an abundant component of their thick mucus, a study reports. These findings shed light on the underlying mechanisms of the…

The United States Adult CF Association (USACFA) is accepting applications to award a total of $15,000 in scholarships to four people with cystic fibrosis (CF)pursuing degrees in the creative arts and other fields. Two of the scholarships — worth $5,000 each — are part of the USACFA’s…

Work targeting new therapies against antibiotic-resistant infections, led by West Virginia University (WVU) researchers, received a $1.4 million grant from a branch of the U.S. Department of Defense (DOD). The grant, awarded by the Defense Threat Reduction Agency, will be used to identify the best combinations of antibodies and…

Researchers have developed a patient-derived model of cystic fibrosis (CF) to better understand whether these patients are more susceptible to COVID-19 infection and its associated serious complications. The project is being led by Ruobing “Ruby” Wang, MD, who cares for CF patients in the division of pulmonary medicine…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…

ELX-02 restored the levels and function of CFTR — the defective protein in CF — in “mini-organs,” or organoids, derived from patients carrying the most common CF-causing nonsense mutation, a study shows. “ELX-02 increased CFTR activity in a dose-dependent fashion,” with these functional increases being “similar to those obtained…

Cystic fibrosis (CF) patients and carriers — those with mutations in only one copy of the CFTR, the gene associated with CF — of European ancestry are at a higher risk of developing pneumonia, according to a U.S. study of genetic risk for this lung disease. CF patients were also…

Limits placed on social interaction by the COVID‐19 pandemic appear to have led to a drop in the number of pulmonary exacerbations — periods of a sudden worsening in lung health — in children with cystic fibrosis (CF), according to a small U.S. study. This finding — in children ages 2 to…

The U.S. Food and Drug Administration (FDA) has expanded the number of cystic fibrosis (CF)-related mutations considered responsive to and therefore eligible for Vertex Pharmaceuticals‘ three approved therapies. With these approvals, the labels will be expanded for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor), and…