The Cystic Fibrosis Foundation (CFF) awarded up to $3.75 million to Matinas BioPharma to support preclinical studies of MAT2501, a potential oral antibiotic to treat nontuberculous mycobacterium (NTM) infections in people with cystic fibrosis (CF) and other lung diseases. A goal of this early research work is…
Using pig lungs as a model, scientists discovered that Staphylococcus aureus, a species of bacteria often found in the lungs of children with cystic fibrosis (CF), preferentially colonizes the mucus circulating there rather than organ tissue itself. According to the researchers, these findings may open new ways of treating…
In an effort to address racism within the cystic fibrosis (CF) community and the health disparities it fosters, the Cystic Fibrosis Foundation (CFF) is asking for greater input from people of color with CF. “We feel great urgency to confront the challenges facing people of color across the…
AzurRx BioPharma is requesting a new patient arm be added to its ongoing Phase 2b trial investigating MS1819 capsules as a treatment for exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF). The request, filed with the U.S. Food and Drug Administration (FDA), would add a patient group…
Adult age up to 50 and chronic antibiotic use seem to be the greatest risk factors for lung infection by the fungus Aspergillus fumigatus among cystic fibrosis (CF) patients, according to a study in Germany. The study, “Risk factors for respiratory Aspergillus fumigatus in German…
A modified version of Pulmozyme (dornase alfa), a widely-used cystic fibrosis (CF) therapy, lasts longer and reduces the need for treatment from once a day to once a week, a study in mice suggests. The study, “PEGylation of Recombinant Human Deoxyribonuclease I Provides a Long‐Acting Version of…
Relizorb, Alcresta Therapeutics‘ pancreatic enzyme treatment, led to significant weight and height improvements among cystic fibrosis (CF) patients with pancreatic insufficiency who require tube feeding, according to the results of a 12-month observational study. The analysis of that study, “Evaluation of the Effectiveness…
Following yearlong advocacy efforts, the triple-combination therapy Trikafta is expected to soon come up for approval before Health Canada, the country’s health regulatory agency, the non-profit patient group Cystic Fibrosis Canada has announced on its website. Vertex Pharmaceuticals, which developed and markets Trikafta, its newest modulator therapy for cystic fibrosis and…
New findings from animal studies support the safety and effectiveness of KB407, a potential inhaled gene therapy for cystic fibrosis, its developer, Krystal Biotech, announced. Preclinical work is ongoing, and the therapy is expected to enter clinical testing in patients by mid-2021. “I am particularly encouraged by progress with KB407 for…
Patient enrollment and dosing has resumed in a Phase 1/2 clinical trial testing MRT5005, Translate Bio‘s potential messenger RNA (mRNA) therapy for people with cystic fibrosis (CF) regardless of their underlying mutation. The trial, called RESTORE-CF trial (NCT03375047) and enrolling up to 40 adults with stable disease at 17 centers…
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