Health Canada is reviewing an application that seeks the approval of Trikafta, a triple combination reported to treat 90% of all with cystic fibrosis (CF), or those with least one F508del mutation. The request from Trikafta’s developer, Vertex Pharmaceuticals, covers eligible patients ages 12 and older, and has been granted…
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Arcturus Therapeutics has selected ARCT-032, an aerosolized RNA-based therapy, to be developed as a novel therapeutic candidate for cystic fibrosis (CF). The therapy will use the company’s LUNAR delivery technology that comprises a library of more than 150 proprietary fatty molecules specifically designed to deliver RNA-based therapies…
Inhaled antibiotic tobramycin safely eradicated early Pseudomonas aeruginosa infection in cystic fibrosis (CF) patients younger than a year old, according to findings from a small retrospective study. The study, “…
The U.S. Food and Drug Administration (FDA) has expanded the number of cystic fibrosis (CF)-related mutations considered responsive to and therefore eligible for Vertex Pharmaceuticals‘ three approved therapies. With these approvals, the labels will be expanded for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor), and…
Scientists discovered the subtypes of gentamicin — a broad-spectrum antibiotic often used to treat bacterial infections in people with cystic fibrosis (CF) — that are more likely to cause deafness, and created a new formulation that is as effective at eliminating bacteria but much safer for patients. “We’ve developed…
The U.K. Cystic Fibrosis Trust is partnering with the U.S. Cystic Fibrosis Foundation to fund a Strategic Research Centre (SRC) — a virtual collaboration bringing together investigators with different backgrounds and areas of expertise — to advance the development of gene-editing therapies for treating cystic fibrosis…
Project GAIA, establishing a large-scale manufacturing process for Pyocin S5, an investigational antibiotic for the treatment of lung disease in people who have cystic fibrosis, is now complete. The scale-up process for Pyocin S5 manufacturing was performed by CPI, an independent technology innovation center in the U.K. This work will…
A newly developed antibiotic, called T405, appears to effectively kill Mycobacteroides abscessus, a difficult-to-treat bacteria that can cause serious infections in people with lung conditions like cystic fibrosis (CF). The antibiotic was described in the study “Development of a penem antibiotic against Mycobacteroides abscessus,” published in Communications…
The Cystic Fibrosis Foundation has awarded a total of $1.7 million in nine grants to research projects focused on tools and strategies to advance therapies for all cystic fibrosis (CF) patients, regardless of their underlying mutations. “There has been an explosion of scientific progress in novel technologies with…
The Cystic Fibrosis Foundation (CFF) has awarded $2.76 million to fund 11 laboratory studies exploring the effects of COVID-19 on people with cystic fibrosis (CF). The hope is that a better understanding of why differences in COVID-19 severity exist among people with CF will ultimately lead to…
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