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…
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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.
A long-term clinical study showed that RELiZORB improves fatty acid absorption in patients suffering from fat malabsorption or pancreatic deficiencies, which is good news for cystic fibrosis (CF) patients suffering from insufficient lipase production. Alcresta Therapeutics, RELiZORB’s developer, presented data from its ASSURE study at the North American Cystic Fibrosis…
Vertex Pharmaceuticals, a clinical-stage biopharmaceutical company, recently announced that two Phase 3 studies evaluating its tezacaftor/ivacaftor combination therapy have shown statistically significant and clinically meaningful improvements in lung function in patients with certain mutations associated with cystic fibrosis (CF). The first study, called EVOLVE (NCT02347657), was conducted to…
Two preclinical studies support the ability of Synspira’s lead candidate SNSP113 to prevent bacteria from forming treatment-resistant layers (biofilms), and in reducing mucus viscosity and easing clearance, new data show. These findings, presented at the 31st Annual North American Cystic Fibrosis Conference (NACFC) taking place in Indianapolis through…
Corbus Pharmaceuticals recently announced that the U.S. Patent and Trademark Office has issued a patent for the use of anabasum, the company’s lead therapy candidate for the treatment of inflammatory and fibrotic diseases. Anabasum is a synthetic oral endocannabinoid-mimetic drug that binds to a receptor called cannabinoid receptor…
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