News

Proteostasis Therapeutics is expecting clinical trial results on three of its cystic fibrosis therapies in the next few months, and plans to start additional trials in 2018. CF is a genetic disease caused by mutations in the sequence of the CFTR gene, which leads to impaired activity of the…

Spyryx Biosciences recently presented preclinical data supporting the clinical benefits of its cystic fibrosis (CF) treatment candidate SPX-101, intended to improve lung function in all patients regardless of their underlying disease-causing mutation. The data, presented in four posters at the recent North American Cystic Fibrosis Conference (NACFC) in Indianapolis,…

News that you’ve just been rejected for a necessary double-lung transplant rarely brings out the best in people. But Caleigh Haber, 27, a self-described fighter since being diagnosed with cystic fibrosis at birth, simply “wasn’t ready to wave the white flag,” she said in an interview with Cystic Fibrosis News…

More adults than children are living with cystic fibrosis for the first time ever, says the Cystic Fibrosis Foundation (CFF). This means more patients are encountering issues they have never had to face — such as saving for their children’s college and their own retirement. They are also having to deal…

Italian researchers have discovered a molecule that prevents cystic fibrosis patients’ anti-inflammatory and anti-bacterial defenses from working as well as they should. Scientists may be able to target the molecule, microRNA-181, to develop CF therapies. The team published their study in the journal Nature. The title is  “MicroRNA-181b…

The European Union’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive recommendation for Orkambi (ivacaftor/lumacaftor) as a therapy for cystic fibrosis (CF) in children. The ruling is specifically for pediatric patients aged 6 to 11 with two copies of the F508del mutation in the…

BIOC51, a potential nitric oxide-based treatment of chronic pulmonary infections due to Pseudomonas aeruginosa bacteria in patients with cystic fibrosis (CF),was awarded special status to promote and speed its development, Novoclem Therapeutics announced. BIOC51 was granted what is known as Qualified Infectious Disease Product (QIDP) status by the U.S. Food and Drug Administration (FDA), whose incentives include priority review…

Vertex Pharmaceuticals announced it will double the number of “All in for CF Scholarships” this year to help more people with cystic fibrosis (CF) and their families. This increase in number of scholarships, the company said, is due to the positive response the program received last year when it was…

Proteostasis Therapeutics is eliminating 13 research positions in order to save $3 million as it “readjusts priorities to focus resources on researching and developing” its cystic fibrosis (CF) programs, including clinical trials for PTI-428, PTI-801 and PTI-808 small molecules. The R&D shift also includes its discovery-stage program in…

Vertex Pharmaceuticals recently presented data from several clinical trials of cystic fibrosis treatments showing positive outcomes for CF patients. Data focused on the approved therapies Orkambi and Kalydeco, and investigative treatments like tezacaftor/ivacaftor and several triple regimens. These findings were presented at the North American Cystic Fibrosis Conference (NACFC) that ran Nov.