Krystal Biotech is expecting to start a Phase 1 clinical trial by early fall to test KB407 for cystic fibrosis (CF), after the investigational inhaled gene therapy was found safe and well-tolerated in nonhuman primates. According to Krystal, the positive…
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Levels of neutrophil elastase, a pro-inflammatory enzyme normally released by immune cells known as neutrophils, plus a greater diversity and amount of microbes could drive disease severity in cystic fibrosis (CF), a study in two siblings suggests. The study, Two Siblings Homozygous for F508del-CFTR Have Varied…
Routine evaluations of how cystic fibrosis (CF) patients and their families are coping with the burden of therapeutic regimens — usually daily therapies that are complex and time-consuming — are an important tool to enhance treatment adherence and create a strong working relationship between patient and care provider, a…
A bioengineer at Rice University will use a more than $2 million federal grant for a project to “repair” harmful mutations that cause cystic fibrosis (CF) using a potentially more accurate approach to gene editing developed in her lab, the university announced in a press release. The National…
To mark its 10th anniversary, Claire’s Place Foundation has launched a website that seeks to connect the community of people affected by cystic fibrosis (CF). “Our new website is a supportive forum for inspiration as well as a resource to learn about developments in CF, our programs and…
A soft skin sticker that can rapidly measure chloride levels in sweat, which are elevated in cystic fibrosis (CF) patients, may help avoid delays in diagnosis and treatment associated with current testing methods, especially in young children, a study reported. The so-called sweat…
An ex vivo technique using nasal epithelial cells from a young girl carrying a rare mutation profile for cystic fibrosis (CF) was successful at predicting the efficacy of an approved CF therapy, a case report shows. Researchers first tested the effectiveness of Orkambi (lumacaftor/ivacaftor) on the child’s own…
Respinova’s non-invasive Pulsehaler, a medical device meant to ease breathing in patients with airway restrictions, such as in cystic fibrosis (CF), has been cleared for marketing in the U.S. Pulsehaler was granted 510(k) clearance from the U.S. Food and Drug Administration (FDA) following the company’s submission of a…
A Phase 2 clinical trial assessing the safety and efficacy of BX004 — BiomX’s phage therapy candidate for Pseudomonas aeruginosa infections in people with cystic fibrosis (CF) — remains on track to have results by the end of this year, the company announced. While this timeline had been…
A clinical trial underway in Israel is investigating how well a personalized chest physiotherapy system compares with traditional airway clearance techniques in people with cystic fibrosis (CF) and other chronic lung conditions, a company reports. “This trial brings us one step closer to our goal: improving the lives of millions…
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