Virus Infecting Common Bacteria Linked to Accelerated Lung Decline in CF
Cystic fibrosis patients with elevated levels of bacteria infected by Pf phage in their sputum experience a faster decline in lung function.
Category: Cystic fibrosis
Cystic fibrosis
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Virus Infecting Common Bacteria Linked to Accelerated Lung Decline in CF
Cystic fibrosis patients with elevated levels of bacteria infected by Pf phage in their sputum experience a faster decline in lung function.
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CF Foundation Invests $15M in ReCode to Develop Gene Editing Therapy
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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How to Help Patients Adhere to Airway Clearance Therapy at Home
Adherence to airway clearance therapy in the home is crucial for managing chronic lung conditions, but challenges like time constraints and lack of perceived benefit can hinder patient compliance. Experts offer strategies and new technologies to help improve patients’ adherence.
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FDA Awards Rare Pediatric Disease Designation to Investigational Cystic Fibrosis Treatment
ETD001 aims to improve lung function by targeting mucus clearance in patients with high unmet medical needs.
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FDA Greenlights Phase 2 Study of Inhaled mRNA Treatment for Cystic Fibrosis
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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First Patients Dosed in Two Phase 1 Trials for Novel Cystic Fibrosis Treatments
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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Most Children with CF Achieve Normal Lung Function with Triple Combination Therapy, Study Finds
An observational one-year study found the therapy significantly reduced lung disease severity and prevented disease progression in children aged 6 to 11 with cystic fibrosis.
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Vertex’s Next-in-class Triple Combo Therapy for Cystic Fibrosis Gets FDA Priority Review
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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Scientists Correct Cystic Fibrosis Genes in Mice for Almost Two Years
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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FDA Fast Tracks Investigational Cystic Fibrosis Drug
SPL84, an antisense oligonucleotide treatment for cystic fibrosis patients with a specific mutation, gains FDA Fast Track designation.
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Liquid Crystal Nanoparticles Supercharge Antibiotics for Cystic Fibrosis
Researchers are developing liquid nanoparticles to bypass the barriers posed by thick mucus and frequent infections in cystic fibrosis lungs and prevent antimicrobial resistance.
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Ivacaftor Safe in CF Infants Aged 1 to 4 Months
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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Jumbo Phage Identified with Potential to Combat CF Lung Infections
Researchers in the United Kingdom have isolated a jumbo phage from wastewater with the potential to fight lung infections caused by Burkholderia cenocepacia.
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SpliSense Gets FDA Nod for Phase 2 Trial for Investigational CF Treatment
The trial will test the efficacy of SpliSense’s inhaled antisense oligonucleotide drug as a potential treatment for the lungs of people with cystic fibrosis with a certain splicing mutation.Â
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Zinc Manipulation Could Restore Immune Defense in Cystic Fibrosis
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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Cystic Fibrosis Foundation Funds Prime Medicine’s Gene Editing Research
The Cystic Fibrosis Foundation will provide up to $15 million to Prime Medicine for preclinical research into gene editing for cystic fibrosis.Â
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Health Canada OKs Ivacaftor for Cystic Fibrosis Patients Down to 2 Months
Health Canada granted market authorization for the expanded use of ivacaftor to treat cystic fibrosis in certain children ages 2 months and older.
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Investigational mRNA Cystic Fibrosis Treatment Granted Orphan Drug DesignationÂ
The drug candidate utilizes a lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs.
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Lab-Made Phlegm Paves Way for Better Understanding of Cystic Fibrosis
Scientists have engineered a living material resembling human phlegm, which will help them to better understand how a certain kind of infection develops on the lungs of patients with cystic fibrosis.
Read MoreHealth Canada OKs Trikafta for Younger Children With Cystic Fibrosis
Health 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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Synthetic Molecules ‘Unclog’ Mucus in Cystic Fibrosis Lungs
Synthetic molecules allowed previously-blocked chloride ions to create 50% more mucus-clearing liquid in cystic fibrosis cells.
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Disease-causing Stem Cells Dominate Lungs of Advanced CF Patients
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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Cystic Fibrosis mRNA Therapeutic Candidate Gets Funding BoostÂ
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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CFTR Modulators Redefining Cystic Fibrosis Care in the Youngest Patients
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.
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