The Cystic Fibrosis Foundation is funding the development of a gene editing therapy that is designed to use ReCode’s tissue-specific delivery vehicle to transport gene editing cargo to the lung cells in people with cystic fibrosis.
The Cystic Fibrosis Foundation is funding the development of a gene editing therapy that is designed to use ReCode’s tissue-specific delivery vehicle to transport gene editing cargo to the lung cells in people with cystic fibrosis.
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The study will evaluate the safety and tolerability of an inhaled peptide therapy designed to enhance CFTR function, act as a bronchodilator, and reduce inflammation.
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The phase 2 study will evaluate the safety and efficacy of ARCT-032, an inhaled mRNA therapy for cystic fibrosis patients, which previously received Orphan Drug Designation.
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The phase 1 trials are evaluating two NBD1 stabilizers, SION-719 and SION-451, which are designed to improve CFTR protein function in cystic fibrosis patients.
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The drug candidate “raises the high bar set by Trikafta,” according to Vertex Pharma’s executive vice president and chief regulatory and quality officer.
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The scientists developed nanoparticles that successfully edited the disease-causing gene in the lungs of a mouse model of cystic fibrosis.
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SPL84, an antisense oligonucleotide treatment for cystic fibrosis patients with a specific mutation, gains FDA Fast Track designation.
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A phase 3 clinical trial, ivacaftor (Kalydeco, Vertex Pharma) was generally safe to administer to infants aged 1-month to <4-months old, according to research published in Journal of Cystic Fibrosis.
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University of Queensland researchers have discovered a fault in the bacteria-killing function of immune cells in people with cystic fibrosis and a potential way to get around it.
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The Cystic Fibrosis Foundation will provide up to $15 million to Prime Medicine for preclinical research into gene editing for cystic fibrosis.Â
Read MoreHealth Canada granted market authorization for the expanded use of Trikafta to include children with cystic fibrosis ages 2-5 years who have at least one copy of the F508del mutation in the CFTR gene.
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Five lung stem cell variants dominate the lungs of patients with advanced cystic fibrosis (CF), and these variants drive key aspects of CF pathology including inflammation, fibrosis, and mucin secretion, according to new research.
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The Cystic Fibrosis Foundation is investing up to $9 million in additional funds in Arcturus Therapeutics to test ARCT-032, an inhaled messenger RNA therapeutic candidate that could treat the underlying cause of cystic fibrosis in all people living with the disease.
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Clinicians share best practices for implementing CFTR modulators in some of their youngest patients, while research explores their potential to lessen the burden of care.
Read MoreA newly-developed cystic fibrosis urine test can make it much easier for doctors to diagnose how badly a patient is affected by the hereditary diseases.
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The combined use of patient-reported symptom data and objective lung function measures may help manage acute respiratory events in pediatric CF patients.
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Using a model reproducing a respiratory epithelium, teams from the University of Geneva have discovered that a film of liquid is sufficient to restore the airways’ seal and lower the risk of bacterial infection.
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The Cystic Fibrosis Foundation announced its first-ever Golden Ticket Competition to find the next promising treatment for cystic fibrosis in a collaboration with Bakar Labs, the incubator at the University of California, Berkeley’s Bakar BioEnginuity Hub.
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A new study has identified hundreds of new proteins that could play a role in cystic fibrosis, and which may provide new insights on why some patients respond better than others to current therapies.
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UNC researchers suggest new targets for cystic fibrosis (CF) therapy and support the case for inflammation as an acquired response unrelated to the CFTR genetic mutation.
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