An exploratory Phase 1 trial showed that repeated intranasal treatment with the therapy eluforsen improved CFTR protein activity in adults with cystic fibrosis (CF) carrying F508del mutations in both CFTR gene copies. The study, “Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis,” was…
News
The World Health Organization (WHO) approved ensifentrine as the recommended name to identify the cystic fibrosis (CF) treatment candidate RPL554. Verona Pharma’s ensifentrine is an inhaled inhibitor of the enzymes phosphodiesterase 3 and 4, designed to have bronchodilator and anti-inflammatory properties. The “-fentrine” staple indicates WHO’s…
An inhaled form of messenger RNA (mRNA) with the potential to safely deliver therapeutics to the lungs of cystic fibrosis (CF) patients has been discovered by researchers at the Massachusetts Institute of Technology (MIT). Researchers developed a type of nanoparticle given as an inhaled mist (nebulizer) that allows the…
Vertex Pharmaceuticals and Arbor Biotechnologies have established a partnership to discover new proteins to advance the development of gene therapies for cystic fibrosis. The companies also plan to develop new genome-editing approaches for four other diseases but haven’t revealed which ones yet. Under the terms of the…
The real estate investment and management firm MC Companies, whose owners are grandparents of children with cystic fibrosis, was recognized for its support of disease research by The Cystic Fibrosis Foundation – Tucson Chapter at its fourth annual Round-Up for a Cure event. Round-Up for a Cure is a yearly fundraising event, put…
As a clinician, I have consulted with many patients over the years and have had the opportunity to be inspired and impacted by many. Most recently I had the pleasure of connecting with Brad, who shared with me his hero-like journey with cystic fibrosis. What struck me the…
AzurRx BioPharma announced it has started a Phase 2 clinical trial testing the safety, tolerability, and efficacy of MS1819 Spray Dried (MS1819-SD) therapy in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI). The trial was approved in October by the U.S. Food and Drug Administration. Many…
Researchers tested blood samples from cystic fibrosis (CF) patients and identified distinct gene expression patterns that could lead to patient-tailored treatments, a study reports. The study, “Identification of molecular signatures of cystic fibrosis disease status using plasma-based functional genomics,” was published in Physiological Genomics. To better understand…
Vast Therapeutics announced that it received funding from the Cystic Fibrosis Foundation (CFF) to support further development of BIOC51, the company’s investigative treatment for antibiotic-resistant bacteria, including Pseudomonas aeruginosa, that is often the cause of lung infections in cystic fibrosis (CF) patients. P. aeruginosa infections set in when mucus accumulation…
Purdue University researchers have invented a new way of delivering two antibiotics (colistin and ciprofloxacin) deep into the lungs of cystic fibrosis (CF) patients, enabling much more effective killing of antibiotic-resistant bacteria without exposing patients to high systemic doses of these therapies. “We are providing a promising option to fight…
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