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Corbus Pharma (CRBP) Reports Agreement with FDA on Phase 2b Cystic Fibrosis Study Design with Pulmon

Multi-national Phase 2b is designed to provide clear evidence of clinical benefit of lenabasum (formerly known as anabasum) in patients with CF Study will enroll ~415 participants with CF 12 years of age and older regardless of underlying CFTR mutation, infection, or background medications First patient expected to be dosed this quarter Management to host conference call and webcast on Tuesday, January 30th at 10:30 a.m. EST Corbus Pharmaceuticals Holdings, Inc. (NASDAQ: CRBP) ("Corbus" or the "Company"), a clinical stage drug development company targeting rare, chronic, serious inflammatory and fibrotic diseases, today announced that it has reached agreement with the U.S. Food and Drug Admi

Gene editing – and what it really means to rewrite the code of life

We now have a precise way to correct, replace or even delete faulty DNA. Ian Sample explains the science, the risks and what the future may hold So what is gene editing? Scientists liken it to the find and replace feature used to correct misspellings in documents written on a computer. Instead of fixing words, gene editing rewrites DNA, the biological code that makes up the instruction manuals of living organisms. With gene editing, researchers can disable target genes, correct harmful mutations, and change the activity of specific genes in plants and animals, including humans. What’s the point? Much of the excitement around gene editing is fuelled by its potential to treat or prevent huma

ProQR and Galapagos Collaborating on a New Way of Developing Fibrosis Treatments

ProQR Therapeutics and Galapagos are working together on a new approach to treating the tissue scarring seen in cystic fibrosis. The collaboration will take advantage of ProQR’s proprietary next-generation RNA technology to develop axiomer editing oligonucleotides that can fight the fibrosis-promoting agents Galapagos has identified. “In this collaboration we will explore a novel application of our Axiomer technology to Galapagos’ fibrosis targets,” Daniel A. de Boer, the CEO of ProQR, said in a press release. “We look forward to working closely with the team at Galapagos. With their leadership in the discovery of novel drug targets and our platform technology and know-how, we can make a mea

Bacterial changes early in life cause cystic fibrosis in kids

Bacterial changes begin in the first few years of life leading to the formation of cystic fibrosis, which makes children repeatedly prone to bacterial infections and inflammation and shortens their lifespan, says a study. Cystic fibrosis is an inherited life-threatening disorder that damages the lungs and digestive system -- the pancreas, liver, kidneys and intestines. Therapies to break up mucus in toddlers could postpone the increase in bacterial burden as well as offer the best route to a longer life for such patients, the researchers said. "Lung symptoms in kids with cystic fibrosis are likely due to an increased burden of bacteria. This implies there's an opportunity for early intervent

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Cystic Fibrosis and Stem Cell Therapy

Cystic fibrosis is a genetic disease that leads to the destruction and damage to the digestive system. It also affects other organs of the body, such as the renal system, liver, and the intestine. Chronic symptoms of this disease include pulmonary infections, difficulty breathing, and the productive cough due to the infection of the lungs by microorganisms. Other signs and symptoms of this disease include fatty steatorrhea, infertility in males. People often present with different kinds of symptoms. This disease can be inherited from one’s parents. It’s an autosomal recessive genetic disease. It’s due to a defective gene, known as the CTFR gene. Cystic fibrosis has no cure, however, treatme

Humanigen’s Inflammation Therapy Is Only Modestly Effective in CF Patients with an Infection, Trial

Humanigen’s anti-inflammatory therapy KB001-A is safe but only modestly effective in cystic fibrosis patients with a Pseudomonas aeruginosa bacteria infection, a Phase 2 clinical trial indicates. In addition to displaying limited ability to decrease markers of patients’ lung inflammation, it failed to generate substantial decreases in their bacteria levels or improvements in their lung function, researchers said. The therapy works by trying to prevent bacteria from releasing toxins that generate inflammation. Researchers’ study, “KB001-A, a novel anti-inflammatory, found to be safe and well-tolerated in cystic fibrosis patients infected with Pseudomonas aeruginosa,” was published in the Jour

Review Board Recommends Continuation of Phase 3 Trial of Anthera’s CF-related Digestive Therapy

An independent review board has given a green light to Anthera Pharmaceuticals continuing a Phase 3 clinical trial evaluating Sollpura’s(lipromatase’s) ability to treat a cystic fibrosis-related digestive disorder. It was the second time that the Data Monitoring Committee had decided that interim findings were good enough for the RESULT trial (NCT03051490) to continue. The study is comparing Sollpura’s and the standard treatment Pancreaze’s ability to treat exocrine pancreatic insufficiency. The committee consists of experts appointed by the Cystic Fibrosis Foundation’s Therapeutics Development Network. The second positive review “of the RESULT study represents one of the last clinical [tria

Preventing Bacteria from Communicating Could Make CF Therapies Like Kalydeco More Effective, Study R

Preventing bacteria from communicating with each other may be a way to bolster the effectiveness of current cystic fibrosis (CF) therapies, a study reports. According to researchers at the University of Montreal Hospital Research Centre (CRCHUM), the use of quorum-sensing inhibitors can help to prevent the harmful effects of bacteria like Pseudomonas aeruginosa, a common source of infection in CF patients, while preserving and enhancing the effects of CFTR corrector drugs (a defective CFTR gene is the cause of CF). These findings were reported in the study “Quorum Sensing Down-Regulation Counteracts the Negative Impact of Pseudomonas aeruginosa on CFTR Channel Expression, Function and Rescue

American CF Patients Now Healthier Overall Than Their Canadian Counterparts, Study Finds

A recent study found improvements in lung function and body mass index (BMI) among children and young adults with cystic fibrosis (CF) both in the United States and Canada from 1990 to 2013, though the American patients improved faster than those in Canada. The study, “Comparison of Nutrition and Lung Function Outcomes in Patients with Cystic Fibrosis Living in Canada and the United States,” appeared in the American Journal of Respiratory and Critical Care Medicine. The analysis — which included data from 5,149 patients in the Canadian Cystic Fibrosis Registry and 37,772 patients in the U.S. Cystic Fibrosis Foundation Registry — compared changes in lung function and BMI among CF patients in

AIT’s Nitric Oxide Mist Lowers CF Patients’ Airway Bacteria by 65%, Phase 2 Trial Shows

AIT Therapeutics’ nitric oxide mist led to a 65 percent reduction in cystic fibrosis patients’ airway bacteria and an improvement in their exercise capability 60 days after treatment, a Phase 2 clinical trial shows. The bacteria that the nitric oxide countered was the Mycobacterium abscessuscomplex. The exercise-capability yardstick researchers used was the 6-minute walk test — or how far patients could walk in 6 minutes. “We continue to be delighted with the data from this trial,” Steve Lisi, chairman and chief executive officer of AIT Therapeutics, said in a press release. “With just 21 days of treatment, patients are still benefitting at Day 81 as shown by 6-minute walk data. Additionally

New Combination Therapy for Cystic Fibrosis

A new combination of cystic fibrosis transmembrane conductance regulator (CFTR) modulators, tezacaftor and ivacaftor, proved effective and safe in treating patients 12 years or older with CFTR gene mutations, according to 2 trials recently published in the New England Journal of Medicine. Tezacaftor, an investigational CFTR corrector, increases CFTR cell surface expression. Ivacaftor, an FDA-approved CFTR potentiator, enhances CFTR channel function. In the first trial, 509 patients homozygous for the most common CFTR mutation that impairs CFTR cell surface expression, Phe508del, were randomly assigned to receive a combination of tezacaftor plus ivacaftor (100 mg of tezacaftor once daily and

Lung Bacteria in CF Children Is Present and Increases in Early Years of Life, Study Finds

Changes in bacteria present in the lower respiratory tract during the first years of life may be indicative of early progression of lung disease in cystic fibrosis (CF) patients, according to researchers at the University of North Carolina School of Medicine. The finding was reported in the study, “Initial acquisition and succession of the cystic fibrosis lung microbiome is associated with disease progression in infants and preschool children,” published in the journal PLoS Pathogens. “Lung symptoms in kids with CF are likely due to an increased burden of bacteria,” Matthew Wolfgang, PhD, associate professor of microbiology and immunology at UNC Chapel Hill and senior author of the study, sa

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