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The Chief Executive of the Cystic Fibrosis Trust recently wrote a letter to the NHS England‘s National Director for Commissioning Operations to help ensure that budget responsibility and delivery of cystic fibrosis (CF) services are not shifted to local commissioners. Ed Owen expressed his concern…

Arcturus Therapeutics, Inc. recently presented encouraging non-human primate studies concerning its preclinical messenger RNA therapies for the potential treatment of genetic diseases such as cystic fibrosis at the 2nd International mRNA Health Conference held November 11-12 in Cambridge, Massachusetts. Arcturus’ presentation, “Potent Delivery of LUNAR™ Nanoparticles Containing Synthetic mRNA for…

New ways of treating cystic fibrosis are under development at the University of Dundee. Along with colleagues in Italy, France, and Scotland, Dr. Anil Mehta, a clinical researcher at Dundee’s Medical Research Institute, pioneered a new drug combination that gives hope to patients with cystic fibrosis. “This work shows…

All over the country and throughout the world, local communities organize events to raise both awareness and funds to help patients suffering with cystic fibrosis (CF), as well as to support research dedicated to finding a cure for the life-threatening genetic disease. Approximately 70,000 people suffer from CF worldwide, 30,000 of which…

In a recent study entitled “Subnanometre-resolution electron cryomicroscopy structure of a heterodimeric ABC exporter” published in the online issue of Nature, the authors determined the structure of an ABC transporter protein with a sub nanometer resolution for the first time. The new findings has implications in…

Massachusetts-based Vertex Pharmaceuticals, Inc. announced yesterday that they have filed for a New Drug Application (NDA) with the US Food and Drug Administration, and a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for their pipeline combination lumacaftor–ivacaftor drug, indicated for the…

Developmental stage biopharmaceutical company Parion Sciences recently announced that the National Institutes of Health‘s grants over the next 5 years in support of pulmonary research programs using their novel pipeline treatments will reach a total of about $15.6 million. These grants have gone to the University of North Carolina,…