CF Foundation Launches New Collaboration with Deep Science Ventures to Overcome Challenges to Developing Genetic Therapies for CF

CF Foundation announced a new collaboration with venture creator Deep Science Ventures, focused on uncovering and designing new technologies with the potential to overcome challenges to developing genetic therapies for cystic fibrosis.
[CF Foundation (BuisnessWire, Inc.)]
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Self-Renewal and Differentiation of Rat Epididymal Basal Cells Using a Novel In Vitro Organoid Model

The authors isolated basal cells from the rat epididymis and established 3-dimensional cell cultures from the basal and non-basal cell fractions. Organoids were formed by single adult epididymal basal cells.
[Biology of Reproduction]
Pinel, L., & Cyr, D. G. (2021). Self-renewal and differentiation of rat Epididymal basal cells using a novel in vitro organoid model. Biology of Reproduction, ioab113. https://doi.org/10.1093/biolre/ioab113 Cite
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AzurRx BioPharma Announces Positive Interim Data 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 positive interim data from the first 18 out of 20 patients 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. (Globenews Wire, Inc.)]
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Vertex Announces European Commission Approval for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Cystic Fibrosis Patients 12 Years and Older With At Least One F508del Mutation in the CFTR gene

Vertex Pharmaceuticals, Inc. announced that the European Commission has granted approval of the label extension for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis in all patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
[Vertex Pharmaceuticals, Inc.]
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Role of Pirfenidone in TGF-β Pathways and Other Inflammatory Pathways in Acute Respiratory Syndrome Coronavirus 2 (SARS-Cov-2) Infection: A Theoretical Perspective

Transforming growth factor-beta (TGF-β), by internalization of the epithelial sodium channel (ENaC), suppresses the anti-oxidant system, downregulates the cystic fibrosis transmembrane conductance regulator (CFTR), and activates the plasminogen activator inhibitor 1 (PAI-1) and nuclear factor-kappa-light-chain-enhancer of activated B cells (NF-kB).
[Pharmacological Reports]
Hamidi, S. H., Kadamboor Veethil, S., & Hamidi, S. H. (2021). Role of pirfenidone in TGF-β pathways and other inflammatory pathways in acute respiratory syndrome coronavirus 2 (SARS-Cov-2) infection: a theoretical perspective. Pharmacological Reports. https://doi.org/10.1007/s43440-021-00255-x Cite
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Transcriptomic Analysis of CFTR-Impaired Endothelial Cells Reveals a Pro-Inflammatory Phenotype

Investigators used an unbiased RNA sequencing approach in complementary models of cystic fibrosis transmembrane conductance regulator (CFTR) silencing and blockade in human endothelial cells to characterise the changes upon CFTR impairment.
[European Respiratory Journal]
Declercq, M., Zeeuw, P. de, Conchinha, N. V., Geldhof, V., Ramalho, A. S., García-Caballero, M., Brepoels, K., Ensinck, M., Carlon, M. S., Bird, M. J., Vinckier, S., Proesmans, M., Vermeulen, F., Dupont, L., Ghesquière, B., Dewerchin, M., Carmeliet, P., Cassiman, D., Treps, L., … Witters, P. (2021). Transcriptomic analysis of CFTR-impaired endothelial cells reveals a pro-inflammatory phenotype. European Respiratory Journal, 57(4). https://doi.org/10.1183/13993003.00261-2020 Cite
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Role of Pirfenidone in TGF-β Pathways and Other Inflammatory Pathways in Acute Respiratory Syndrome Coronavirus 2 (SARS-Cov-2) Infection: A Theoretical Perspective

Transforming growth factor-beta (TGF-β), by internalization of the epithelial sodium channel (ENaC), suppresses the anti-oxidant system, downregulates the cystic fibrosis transmembrane conductance regulator (CFTR), and activates the plasminogen activator inhibitor 1 (PAI-1) and nuclear factor-kappa-light-chain-enhancer of activated B cells (NF-kB).
[Pharmacological Reports]
Hamidi, S. H., Kadamboor Veethil, S., & Hamidi, S. H. (2021). Role of pirfenidone in TGF-β pathways and other inflammatory pathways in acute respiratory syndrome coronavirus 2 (SARS-Cov-2) infection: a theoretical perspective. Pharmacological Reports. https://doi.org/10.1007/s43440-021-00255-x Cite
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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]
Vaidyanathan, S., Baik, R., Chen, L., Bravo, D. T., Suarez, C. J., Abazari, S. M., Salahudeen, A. A., Dudek, A. M., Teran, C. A., Davis, T. H., Lee, C. M., Bao, G., Randell, S. H., Artandi, S. E., Wine, J. J., Kuo, C. J., Desai, T. J., Nayak, J. V., Sellers, Z. M., & Porteus, M. H. (2021). Targeted replacement of full-length CFTR in human airway stem cells by CRISPR/Cas9 for pan-mutation correction in the endogenous locus. Molecular Therapy, 0(0). https://doi.org/10.1016/j.ymthe.2021.03.023 Cite
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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]
Comegna, M., Conte, G., Falanga, A. P., Marzano, M., Cernera, G., Di Lullo, A. M., Amato, F., Borbone, N., D’Errico, S., Ungaro, F., d’Angelo, I., Oliviero, G., & Castaldo, G. (2021). Assisting PNA transport through cystic fibrosis human airway epithelia with biodegradable hybrid lipid-polymer nanoparticles. Scientific Reports, 11(1), 6393. https://doi.org/10.1038/s41598-021-85549-z Cite
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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]
Role of extracellular vesicles in chronic lung disease | Thorax. (n.d.). Retrieved March 18, 2021, from https://thorax.bmj.com/content/early/2021/03/11/thoraxjnl-2020-216370.long Cite
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