News

PP-007 Resolves Lung Inflammation in CF Mouse Model

Treatment with PP-007 — a compound known to promote the release of anti-inflammatory carbon monoxide (CO) gas — significantly reduced the levels of pro-inflammatory immune cells and molecules in the lungs of a mouse model of cystic fibrosis (CF), a study shows. These benefits were associated with an increased production…

Study: Most CF Infants Had Healthy Stools in First Year

Stool consistency and frequency were healthy for the majority of infants with cystic fibrosis (CF) in the first year of life, according to an analysis of data from the BONUS study. Stool consistency and frequency were not associated with pain, while infants exclusively fed with formula or who received…

High Variability Found in People With F508del/5T;TG12 Genotype

There is wide clinical variety in the presentation of individuals who carry one F508del mutation and one 5T;TG12 mutation in the CFTR gene, an Italian study highlights. Its researchers stressed the need to monitor individuals with this genotype, since there are not currently accurate ways to predict who is at highest risk…

Aerosolized Therapy Restores CFTR in Patient-derived Cells

ReCode Therapeutics’ investigational aerosolized RNA-based therapies for cystic fibrosis (CF) restored CFTR function, the defective protein in CF, in patient-derived lung cells. Delivered by the company’s selective organ targeting (SORT) lipid nanoparticle (LNP) platform, the therapy showed no signs of toxicity in epithelial cells that line the airways.