Targeted Replacement of Full-Length CFTR in Human Airway Stem Cells by CRISPR/Cas9 for Pan-Mutation Correction in the Endogenous Locus

Scientists used CRISPR/Cas9 and two adeno-associated viruses carrying the two halves of the cystic fibrosis transmembrane conductance regulator (CFTR) cDNA to sequentially insert the full CFTR cDNA along with a truncated CD19 enrichment tag in upper airway basal stem cells and human bronchial basal stem cells.
[Molecular Therapy]
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AzurRx BioPharma Announces Completion of Enrollment in Phase II Clinical Trial of MS1819 in Combination with PERT in the Treatment of Cystic Fibrosis Patients with Severe Exocrine Pancreatic Insufficiency

AzurRx BioPharma, Inc. announced it has completed enrollment in its Phase II trial evaluating MS1819 in combination with the current standard of care, porcine-derived pancreatic enzyme replacement therapy (PERT), for the treatment of severe exocrine pancreatic insufficiency in patients with cystic fibrosis.
[AzurRx BioPharma, Inc.]
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Assisting PNA Transport through Cystic Fibrosis Human Airway Epithelia with Biodegradable Hybrid Lipid-Polymer Nanoparticles

Investigators loaded hybrid core–shell nanoparticles (hNPs) with a 7-mer peptide nucleic acid (PNA) previously considered for its ability to modulate the post-transcriptional regulation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. They also investigated the in vitro release kinetics of hNPs and their efficacy in PNA delivery across the human epithelial airway barrier using an ex vivo model based on human primary nasal epithelial cells from CF patients.
[Scientific Reports]
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Role of Extracellular Vesicles in Chronic Lung Disease

The authors provide a broad overview of the roles of extracellular vesicles (EV) in chronic respiratory disease. Recent advances in profiling EVs have shown their potential as biomarker candidates. Further studies have provided insight into their disease pathology, particularly in inflammatory processes across a spectrum of lung diseases.
[Thorax]
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LasR-Deficient Pseudomonas Aeruginosa Variants Increase Airway Epithelial mICAM-1 Expression and Enhance Neutrophilic Lung Inflammation

Scientists investigated how lasR mutants modulate airway epithelial membrane bound mICAM-1, a surface adhesion molecule, and determined its impact on neutrophilic inflammation in vitro and in vivo.
[PLoS Pathogens]
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Impact of Hypoxia and Ampk on Cftr-Mediated Bicarbonate Secretion in Human Cholangiocyte Organoids

Scientists investigated the effect of hypoxia on cystic fibrosis transmembrane conductance regulator activity in intrahepatic cholangiocyte organoids and test drug-interventions to restore bicarbonate secretion.
[American Journal of Physiology-Gastrointestinal and Liver Physiology]
Roos, F. J. M., Bijvelds, M. J. C., Verstegen, M. M. A., Roest, H. P., Metselaar, H. J., Polak, W. G., Jonge, H. R. de, IJzermans, J. N. M., & Laan, L. J. W. van der. (2021). Impact of hypoxia and AMPK on CFTR-mediated bicarbonate secretion in human cholangiocyte organoids. American Journal of Physiology-Gastrointestinal and Liver Physiology. https://doi.org/10.1152/ajpgi.00389.2020 Cite
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Hepatobiliary Organoids and Their Applications for Studies of Liver Health and Disease: Are We There Yet?

The authors summarize currently available adult cell‐derived hepatobiliary organoid models and their applications.
[Hepatology]
Shiota, J., Samuelson, L. C., & Razumilava, N. (n.d.). Hepatobiliary organoids and their applications for studies of liver health and disease: are we there yet? Hepatology, n/a(n/a). https://doi.org/https://doi.org/10.1002/hep.31772 Cite
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AzurRx BioPharma Announces Completion of Enrollment in First Cohort of Phase IIb OPTION 2 Extension Study of MS1819

AzurRx BioPharma, Inc. announced the completion of patient enrollment in the first cohort of the Phase IIb OPTION 2 extension study evaluating immediate-release capsules of MS1819 for the treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis.
[AzurRx BioPharma, Inc.]
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AzurRx BioPharma Adds Two New Clinical Trial Sites in Europe for Phase IIb OPTION 2 Extension Study of MS1819 Immediate-Release Capsules in Cystic Fibrosis Patients

AzurRx BioPharma, Inc announced the activation of two additional clinical trial sites in Poland for the extension arm of the Phase IIb OPTION 2 study investigating immediate-release capsules of MS1819 for the treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis.
[AzurRx BioPharma, Inc.]
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Modulation of cAMP Metabolism for CFTR Potentiation in Human Airway Epithelial Cells

Human airway epithelial cells were used to test the efficacy of cyclic AMP modulation by ABCC4 and PDE-4 inhibition through a series of concentration–response studies.
[Scientific Reports]
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Correction of CFTR Function in Intestinal Organoids to Guide Treatment of Cystic Fibrosis

Scientists studied cystic fibrosis transmembrane conductance regulator (CFTR) function in organoids of subjects with common and rare CFTR mutations and evaluated correlations between CFTR function and clinical data.
[European Respiratory Journal]
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