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Preclinical Work Supports ELX-02, Potential Treatment for Nonsense Mutations

Eloxx Pharmaceuticals presented preclinical safety and efficacy data supporting ELX-02, its investigational treatment for cystic fibrosis (CF) caused by nonsense mutations that is also being evaluated in two enrolling, Phase 2 trials. The data were presented as two posters at this year’s virtual North American Cystic Fibrosis Virtual Conference (NACFC). Poster abstracts were also electronically published as a supplement to the journal Pediatric Pulmonology. “We were pleased to have the opportunity to present additional preclinical study results,” Gregory Williams, PhD, CEO of Eloxx, said in a press release. “We believe that these results de-risk the current Phase 2 proof of concept clinical

Cystic fibrosis (CF) and staphylococcus aureus infections

Staphylococcus aureus (known as staph) is a bacterium that is quite common in the general population. If someone with cystic fibrosis (CF) develops a staph infection, the bacteria may grow and spread quickly, causing more serious problems. This page from Great Ormond Street Hospital (GOSH) explains about staph infections and how we will manage them when your child has CF. Staphylococcus aureus (staph) is a bacterium or germ that is commonly found on our skin or in our upper respiratory tract (windpipe). The bacteria can pass from person to person by skin contact but also in droplet form released when someone sneezes or coughs. Reducing the spread It can be difficult to prevent staph infectio

New medical trial for Cystic Fibrosis patients helps with medication side effects

The coronavirus pandemic has had a surprising benefit for cystic fibrosis patients. Dr. Perry Brown, with the St. Luke's Idaho Cystic Fibrosis Center, says they're seeing fewer hospitalized pediatric patients since people are wearing masks and social distancing. A big part of CF is chronic respiratory infections, and the best way to manage that is with antibiotics. However, using these antibiotics can affect the kidneys and hearing for many adult CF patients. Brian Devine has seen many advancements in his 45 years of living with cystic fibrosis. "Back then, there was very little known about cystic fibrosis and very little treatment, and it just progressed drastically over the last several ye

IV antibiotics not superior to oral therapy for eradicating P. aeruginosa in cystic fibrosis

Treatment with IV antibiotics, compared with oral therapy, did not achieve sustained eradication of Pseudomonas aeruginosa in a greater proportion of patients with cystic fibrosis, according to results of the TORPEDO-CF trial. “Current evidence supports starting antibiotic regimens that aim to eradicate P. aeruginosa as soon as the infection is diagnosed to disrupt nascent biofilms and prevent progression to chronic infection,” Simon C. Langton Hewer, MD, consultant pediatrician in the department of pediatric respiratory medicine at Bristol Royal Hospital for Children at the University of Bristol, U.K., and colleagues wrote. “However, evidence to suggest that any one regimen is better than a

Treating cystic fibrosis with mRNA therapy or CRISPR

New Rochelle, NY, October 8, 2020--The potential for treating cystic fibrosis (CF) using mRNA therapies or CRISPR gene editing is possible regardless of the causative mutation. CF clinical trials showing that a genotype-agnostic gene therapy for CF is possible are reviewed in the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free on the Human Gene Therapy website through November 4, 2020. "Treating CF by delivering mRNA that encodes CFTR has the potential to work in any CF patient, independent of the underlying mutation," state James Dahlman, Georgia Institute of Technology, and coauthors. "Another potential treatment is utilizing mRNA encoding nucleases

Certain Factors Could Predict Severe Episodes of Coughing Up Blood in CF Patients, Study Says

Factors such as diabetes, a specific type of fungal infection, and previous mild to moderate episodes of hemoptysis, or coughing up blood, may help predict the risk of massive hemoptysis in people with cystic fibrosis (CF), a study suggests. The study, “Predictors of massive haemoptysis after a first episode of mild-to-moderate haemoptysis in patients with cystic fibrosis,” was published in the European Respiratory Journal. Older age, advanced lung disease, Pseudomonas aeruginosa lung infections, CF-related diabetes, portal hypertension (high blood pressure in the liver’s portal vein), and liver cirrhosis have all been described as risk factors for hemoptysis in CF. But it is still unclear w

Calithera Biosciences’ CB-280 Arginase Inhibitor Trial in Progress Poster Presented at the NACFC

SOUTH SAN FRANCISCO, Calif., Oct. 07, 2020 (GLOBE NEWSWIRE) -- Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical stage biotechnology company focused on discovering and developing novel small-molecule drugs for the treatment of cancer and other life-threatening diseases, today announced that a poster detailing the trial design of a Phase 1b study of CB-280, the company’s arginase inhibitor, is being presented today at the North American Cystic Fibrosis (NACFC) 2020 Virtual Conference. The randomized, double-blind, placebo-controlled, multiple ascending dose-escalation study (NCT04279769) is exploring CB-280 versus placebo in adults with cystic fibrosis and chronic infection with Pseudomo

Lenabasum Fails to Lessen Exacerbations in CF Patients in Phase 2 Trial

A Phase 2 clinical trial of oral lenabasum in people with cystic fibrosis (CF) failed to meet its primary goal of reducing pulmonary exacerbations, the investigational therapy’s developer, Corbus Pharmaceuticals, announced. “We are very disappointed that the study did not meet the primary endpoint,” Barbara White, MD, chief medical officer and head of research at Corbus, said in a company press release. Lenabasum — formerly known as anabasum, JBT-101, or resunab — is a synthetic, small molecule designed to reduce inflammation and limit fibrosis by binding to a receptor present on activated immune cells called cannabinoid receptor type 2 (CB2). Data from a previous Phase 2 clinical trial, CF-

FDA Grants Approval To Ivacaftor For Treatment Of Cystic Fibrosis In Infants

Study did not meet primary endpoint of reducing rate of pulmonary exacerbations Lenabasum treatment had a favorable safety profile and was well-tolerated Data to be presented at the upcoming North American Cystic Fibrosis Conference Oct. 7-23 Norwood, MA, Oct. 06, 2020 (GLOBE NEWSWIRE) -- Corbus Pharmaceuticals Holdings, Inc. (NASDAQ: CRBP) (“Corbus” or the “Company”), a clinical-stage drug development company pioneering transformative medicines that target the endocannabinoid system, today announced topline results from its 28-week Phase 2b study of lenabasum in patients with cystic fibrosis (CF). The study enrolled patients in the U.S., Canada and Europe at high risk for recurrent pulmonar

Oral antibiotics are as effective as IV antibiotics for the eradication of PseudomonasOral antibiot

The results of the long-running 'Torpedo' clinical trial investigating two different approaches to the eradication of a serious cystic fibrosis (CF) lung infection were published this week. They show that oral antibiotics are as effective as intravenous antibiotics for removing the first infection of Pseudomonas aeruginosa. The bacteria Pseudomonas aeruginosa (P. aeruginosa) causes serious lung infections in people with cystic fibrosis. It is important to prescribe antibiotics as early as possible, to remove (‘eradicate’) the infection and prevent it from becoming established in the lungs. Once it becomes established, antibiotics are used to manage the infection and minimise the amount of lu

Study Compares Oral and Intravenous Antibiotics for P. aeruginosa Infections in People With CF

Oral antibiotics are just as effective as intravenous (into-the-vein) antibiotics at killing Pseudomonas aeruginosa bacteria in people with cystic fibrosis (CF), a new study shows. This finding may allow people with CF to spend less time in the hospital to receive treatment, ultimately reducing treatment burden. The study, “Intravenous versus oral antibiotics for eradication of Pseudomonas aeruginosa in cystic fibrosis (TORPEDO-CF): a randomised controlled trial,” was published in The Lancet Respiratory Medicine. Pseudomonas aeruginosa is a type of bacteria that commonly causes problematic lung infections in people with CF. It is estimated that most people with CF develop chronic P. aerugino

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