The first patient has been dosed in a Phase 2 clinical trial evaluating RPL554 as a treatment for cystic fibrosis (CF), according to its maker, Verona Pharma. RPL554 is a first-in-class drug candidate designed to inhibit the enzymes phosphodiesterase 3 and 4 (PDE3/PDE4). It has been shown to have…
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Dutch pharmaceutical company ProQR Therapeutics now has two key patents protecting its first-in-class ribonucleic acid (RNA)-based oligonucleotide QR-010 for the treatment of cystic fibrosis (CF) in the United States and Europe. The patents are valid until at least July 2033. The new U.S. patent, announced April 3, covers methods of targeting…
Corbus Pharmaceuticals recently reported that its Phase 2 clinical trial examining multiple doses of anabasum (formerly known as JBT-101 or Resunab) compared to placebo in patients with cystic fibrosis (CF) achieved the trial’s primary endpoint demonstrating an acceptable safety and tolerability profile at all doses, with no serious or severe adverse…
Vertex Pharmaceuticals recently announced positive results of two Phase 3 clinical trials, the EVOLVE and EXPAND studies, evaluating the effects of a combination of tezacaftor (VX-661) and ivacaftor (sold under the brand name Kalydeco) in cystic fibrosis (CF). The results showed that the new combined therapy significantly improved…
David M. Rodman is the new chief development strategy officer at ProQR Therapeutics, a leader in RNA therapies for cystic fibrosis (CF) and other diseases. Rodman, a medical doctor, will be tasked with advancing ProQR’s treatment candidates. Dr. David M. Rodman. Photo credit: ProQR A key therapy…
Researchers from Queen’s University Belfast in Northern Ireland have not only discovered why antibiotics are becoming less effective at treating infections in cystic fibrosis (CF) patients — but have also found a potential solution: fat-soluble vitamins. Lung infections, mostly those caused by bacteria, are a serious problem for people with CF and are…
Stem cell therapy may reduce lung inflammation in cystic fibrosis (CF) and chronic obstructive pulmonary disease (COPD), according to preliminary results of a pre-clinical-trial study. The findings, presented at this year’s Lung Science Conference in Estoril, Portugal, could lead to new ways to treat the diseases. The title of…
A wide range of genetic diversity in a bacteria common in the lungs of cystic fibrosis (CF) patients may explain why some develop life-threatening infections while others appear unaffected, according to a study. The research, “Phenotypic diversity and genotypic flexibility of Burkholderia cenocepacia during long-term chronic infection…
Mucus proteins that fail to achieve the correct structure cause airway secretions to become thick and sticky in patients with cystic fibrosis (CF), leading to the well-known symptoms of the disease, according to a study. The reason the proteins fail to assume the shape that’s seen in healthy people is simple,…
Rockefeller University researchers have mapped the three-dimensional structure of the protein involved in cystic fibrosis (CF). The mapping is important because knowing more about the CFTR protein will provide scientists with valuable insight into its activity and may lead to novel therapies for CF. Findings of the study, “Molecular…
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