Cempra, Inc received clearance to initiate Phase 1b and Phase 2 / 3 studies that will test their solithromycin antibiotic in newborn to 17 year-old patients. The experimental antibiotic could eventually offer new therapeutic options to cystic fibrosis patients in fighting bacterial lung infections. Intravenous, oral suspension formulas, and…
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Clinical-stage biopharmaceutical company, N30 Pharmaceuticals, recently announced that it raised $30 million in a mezzanine round of financing, which will be used to accelerate the development the company’s investigational therapy for the treatment of cystic fibrosis (CF). The novel drug is called N91115 and N30 Pharma has successfully completed its phase…
The Cystic Fibrosis Foundation (CFF) announced this week that its nonprofit affiliate, Cystic Fibrosis Foundation Therapeutics, has sold its royalty rights for CF treatments developed by Vertex Pharmaceuticals Inc., including the new cystic fibrosis drug Kalydeco. The Foundation sold the rights to Royalty Pharma for…
In a recent study entitled “Modulation of the Maladaptive Stress Response to Manage Diseases of Protein Folding,” a team of researchers showed that in protein misfolding diseases, such as cystic fibrosis and Alzheimer’s disease, the cells’ response to misfolding proteins can actually exacerbate the disease phenotype. The study was published in the …
A project for a tablet-based software tool designed to help children suffering from lung diseases properly use nebulizers, which may be helpful to cystic fibrosis patients (CF), was recently awarded $100,000 at the Pitt Innovation Challenge (PInCh). The device was one of three projects to be awarded with the top prize to fund…
A major breakthrough in the understanding of respiratory diseases entitled “The draft genome sequence of the ferret (Mustela putorius furo) facilitates study of human respiratory disease” was published on Nature Biotechnology by Dr. Xinxia Peng at the University of Washington in Seattle and Federica Di…
Cystic Fibrosis (CF) is caused by mutations in the Cftr gene (short for cystic fibrosis transmembrane conductance regulator). The CFTR protein is responsible for transporting chloride and sodium ions in and out of lung and intestinal epithelium cells, and so in the absence of CFTR functional protein, thick mucus…
A recent paper published in the European Respiratory Journal reports that Canadians suffering with cystic fibrosis (CF) are living nearly 20 years longer than they did two decades ago. In 1990, the median survival age for Canadian patients with cystic fibrosis on average was 31.9 years. That number increased to 49.7…
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…
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