Vertex Pharmaceuticals VRTX announced that the FDA has approved its next-in-class vanza triple therapy for treating people with cystic fibrosis ... regulator (CFTR) gene responsive to the drug.

“ARCT-810 is the only mRNA therapy ... Fibrosis Cystic fibrosis is a life-shortening disease with a worldwide prevalence. Mutations in the cystic fibrosis transmembrane conductance regulator ...

Gene Therapy and the Challenge of Lung Delivery Gene therapy, a promising treatment for genetic disorders like cystic fibrosis ... Their success in delivering mRNA in COVID-19 vaccines has ...

“ARCT-810 is the only mRNA therapy currently ... of OTC deficiency.” Cystic fibrosis is a life-shortening disease with a worldwide prevalence. Mutations in the cystic fibrosis transmembrane ...

A gene therapy based on an enhanced lipid nanoparticle (LNP) loaded with vascular endothelial growth factor (VEGF) mRNA could be developed for the treatment of preeclampsia, according to a study in ...

Because cystic fibrosis affects multiple organ systems, including the lungs and digestive system, people with the condition often need care from a team of healthcare professionals. Cystic fibrosis ...

Uta Griesenbach, Eric W.F.W. Alton, Department of Gene Therapy, Faculty of Medicine ... proof-of-principle for gene transfer into the cystic fibrosis (CF) lung. It is currently unknown whether ...

The FDA has approved Vertex’s Alyftrek, a once-daily triple combination for the treatment of cystic fibrosis in people 6 years and older who have mutations that are amenable to the therapy ...