A new population of lung stem cells capable of repairing the airways following a severe injury was identified in two preclinical studies. These cells’ ability to transform and replace several lung cell types may be explored for regenerative medicines to treat diseases such as cystic fibrosis.
The Therapeutics Development Network (TDN) of the Cystic Fibrosis Foundation has endorsed Proteostasis Therapeutics‘ trial assessing PTI-428 in combination with Symdeko (tezacaftor/ivacaftor) as a treatment for cystic fibrosis (CF). The Phase 2 trial will assess the safety, tolerability and pharmacokinetics of multiple doses of PTI-428 over a 28-day…
Enterprise Therapeutics recently closed a £29 million ($41 million) Series B financing round to help advance into clinical development the company’s pipeline of potential mucus-targeting therapies for cystic fibrosis (CF) and other lung diseases with a high unmet medical need. “We are very encouraged by progress the Enterprise team…
The U.S. Food and Drug Administration has given the go-ahead for MRT5005, Translate Bio’s lead candidate to treat the underlying cause of cystic fibrosis (CF), to be tested in a Phase 1/2 clinical trial. The company expects to begin dosing enrolled patients by mid-year. MRT5005 is designed to address…
Autogenic drainage physiotherapy can effectively improve the underlying lung mechanical impairment of cystic fibrosis (CF), a study shows. Respiratory physiotherapy is an important part of the treatment of patients with CF because it helps remove the mucus that accumulates in the lungs and that can ultimately lead to respiratory failure.
The two components of Orkambi act separately to trigger a liver enzyme that decreases the therapy’s ability to fight cystic fibrosis, a study reported. Vertex Pharmaceuticals created Orkambi by combining ivacaftor and lumacaftor (VX-809). It addresses the underlying cause of the disease — a gene mutation that leads to a build-up of…
Abnormalities in continuous glucose monitoring (CGM) measures correlate with a decline in lung function in children and adolescents with cystic fibrosis (CF), a study suggests. The study, “Continuous glucose monitoring abnormalities in cystic fibrosis youth correlate with pulmonary function decline,” was published in the Journal of Cystic…
Bacteria can transmit memory across generations so their descendants will know how to attach themselves to surfaces and form communities called biofilms, a study reports. Researchers’ findings shed light on cystic fibrosis-related bacteria’s early steps in biofilm formation. These pathogens cause persistent airway infections in people with the disease. The…
The Cystic Fibrosis Foundation (CFF) has joined 70 other organizations in signing a letter opposing the latest version of the Right to Try Act, which the House passed recently. It would allow terminally ill Americans to try medicines that have passed Phase 1 of the U.S. Food and Drug Administration…
The U.S. Food and Drug Administration has granted Fast Track Designation to Proteostasis Therapeutics’ triple combo treatment program for cystic fibrosis. The program includes the company’s amplifier, corrector, and potentiator of the CFTR gene — a combination designed to overcome the impact of the mutations that cause CF. The…
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