News

A new study suggests a method for correcting and improving stem cells derived from people with cystic fibrosis (CF), one that could lead to new ways of treating CF using a patient’s own cells. The report, titled “Robust method for TALEN-edited correction of pF508del in patient-specific induced pluripotent stem cells,“ appeared…

Nivalis Therapeutics, Inc., recently announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead investigational drug, N91115, a stabilizer of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, as a potential treatment for patients with cystic fibrosis (CF). The drug is now being evaluated in…

Galapagos NV announced the start of its SAPHIRA Phase 2 exploratory study with the first dosing of GLPG1837 in a cystic fibrosis (CF) patient. Topline results are expected in late 2016. CF is triggered by a mutation in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein, resulting in abnormal…

Collaborative research between Eindhoven University of Technology (Netherlands), McGill University (Canada), and the University of Duisburg-Essen (Germany) has identified a promising approach that could lead to the development of an efficient treatment for cystic fibrosis (CF). The paper, “Characterization and small-molecule stabilization of the multisite tandem binding between 14-3-3…

Rhinovirus infections in children with cystic fibrosis (CF) are linked to a lack of improvement in lung function after treatment for pulmonary exacerbations caused by the infection, a recent study reported. The study,“Rhinovirus-associated pulmonary exacerbations show a lack of FEV1 improvement in children with cystic fibrosis,“ analyzed 18 children who were…

A study comparing two continuous treatment regimens of tobramycin inhalation (Tobi) for Pseudomonas aeruginosa infection in patients with cystic fibrosis (CF) found that administration of both once a day and twice daily treatment was safe and well-tolerated. The study, titled “Pharmacokinetics and safety of an 8 week continuous treatment with once-daily versus twice-daily inhalation…

Researchers identified a novel, rare mutation causing cystic fibrosis (CF) in a pediatric African-American patient. The mutation, however, was found to be responsive to the CFTR corrector VX-809, therefore identifying potential personalized therapeutics. The study, “c.3623G > A mutation encodes a CFTR protein with impaired channel function,” was published…

The University of Iowa (UI) Research Foundation recently announced it had struck a license and sponsored research agreement with Pfizer to support the development of a potential gene therapy for cystic fibrosis (CF). The research will be performed in the the laboratories of Profs. John Engelhardt and Ziying Yan. Both…

Alegeus recently announced that it will donate a total of $20,000 to three charities — including the Cystic Fibrosis Foundation — as part of its 2015 holiday giving program. Instead of handing out corporate gifts and holiday cards, Alegeus opted to let employees chose charities to support. Votes given to the three…

Vertex Pharmaceuticals Incorporated recently announced that it has received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) to its supplemental New Drug Application (sNDA) for Kalydeco (ivacaftor) as a treatment for cystic fibrosis patients ages 2 and older with one of 23 residual function mutations in the cystic fibrosis transmembrane conductance…