Porosome Therapeutics will patent a new treatment for cystic fibrosis that targets a cell structure called the porosome.
Porosome Therapeutics submitted to register a patent for a new therapy against cystic fibrosis and other diseases. The manufacturer calls the drug the first remedy that uses a porosoma, a cellular structure that is the secretory mechanism of a cell.
Porosomes are lipoprotein structures on the cell’s plasma membrane where secretory vesicles temporarily dock and fuse, releasing intravesicular contents from the cell. Porosome defects can lead to the development of diseases such as neurological and immune disorders, various types of cancer, diabetes and cystic fibrosis. Founder of Porosome Therapeutics Bhanu P. Jena discovered Porosoma more than 20 years ago.
Cystic fibrosis is a genetic disease that causes chronic lung infections due to mucus buildup in the lungs, leading to progressive and potentially fatal lung damage. The cause is a mutation in a gene that codes for a protein called the cystic fibrosis transmembrane conductance regulator (CFTR). Protein regulates the transport of chlorides, sodium and bicarbonates across epithelial membranes.
Unlike Vertex’s Orkambi and Trikafta, Porosome offers two new ways to treat cystic fibrosis. One of them is to use non-invasive methods to introduce a healthy porosome into a diseased cell, which leads to the replacement of the mutated protein and the restoration of mucin secretion and organ functions. In the second variant, small molecules are used to restore the secretory function of the affected proteins in porosomes.
“While there are treatments available to reduce the symptoms and effects of cystic fibrosis, prior to the pioneering development of Porosome Therapeutics, no existing therapies had addressed the mutated CFTR protein in terms of its secretory ecosystem,” said the CEO of Porosome Therapeutics. Guillermo Marmol.
2023-08-25 15:50:40
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