Porosome Therapeutics has announced a potentially novel treatment for cystic fibrosis while stating that it has filed a comprehensive patent for the new therapy in cystic fibrosis and other diseases. The biopharma refers to the new therapy aimed at treating cystic fibrosis (CF) as the first application of the porosome, which is the secretory portal in cells.
Porosomes are lipoproteins at the cell plasma membrane that promote cellular communication by secreting chemical messages, which are important to sustain life. The secretory defects in the porosome are implicated in diseases such as neurological disorders, immune disorders, various cancers, diabetes, and CF.
CF is a genetic disorder that causes chronic lung infections due to the mucus build-up that is thick and sticky and progressive lung damage that could potentially be deadly. It occurs from missing cystic fibrosis transmembrane conductance regulatory (CFTR) proteins caused by mutations in the CFTR gene.
The company announced on 6 August that it was no longer Viron but Porosome Therapeutics to reflect its fundamental research centred on the porosome complex.
Founder and Chairman of Porosome Therapeutics, Professor Bhanu P. Jena said: “By incorporating normally functioning CFTR-associated porosomes into diseased cells or by specifically targeting affected porosome proteins, these proposed therapies will ameliorate cystic fibrosis symptoms at a fundamental level, restoring function and thereby treating the disorder.”
Guillermo Marmol CEO Porosome Therapeutics said: “While treatments exist to lessen symptoms and consequences of cystic fibrosis, until Porosome Therapeutics’ ground-breaking development, none of the existing treatments have addressed the CFTR-mutated protein in terms of its secretory ecosystem.”
“As the discoverer of the porosome and its potential, and following decades of research, Porosome Therapeutics’ founder has inspired its incredible team to unleash the body’s existing yet untapped capabilities of self-generation to effectively address the core cause of cystic fibrosis symptoms.”
Unlike Vertex’s Orkambi (lumacaftor/ivacaftor) and Trikafta (elexacaftor, ivacaftor and tezacaftor), Porosome Therapeutics uses two treatment pathways for CF. One is the use of non-invasive methods to insert a healthy porosome into a diseased cell, replacing the mutated protein and restoring mucin secretion and function in organs.
Another pathway is introducing small molecules to restore secretory function to implicated porosome proteins. Porosome Therapeutics notes that its studies on human lung epithelial cell lines expressing cystic fibrosis have shown significant – and unprecedented – enhancement of mucin secretion.
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