Porosome now seeking US patent to cover new treatment for CF
Company says its platform will launch 'new era of drug development'
Porosome Therapeutics is seeking a U.S. patent that would cover its “radically new and distinctive” technology platform for the treatment of cystic fibrosis (CF) and other health conditions, the company said in a press release. Its platform works by targeting a cellular structure called the porosome.
The global biopharmaceutical, based in Boston, now has filed what it called a comprehensive patent application for its “paradigm-shifting approach.”
“The porosome platform provides a promising entry into a new era of drug development and therapy,” said Bhanu P. Jena, PhD, founder and chairman of Porosome Therapeutics.
The patent, if granted, would cover “the first therapeutic application of the porosome, the cell’s secretory apparatus,” the company stated in the release.
New treatment platform targets the porosome cell structure
CF is caused by mutations in the gene that provides instructions for making the protein CFTR. This protein plays a critical role in producing mucus; in CF, dysfunctional CFTR leads to the production of abnormally thick and sticky mucus, which drives most symptoms of the disease.
The CFTR protein normally sits at the surface of cells and helps to control the flow of water and salts in and out of the cell. Normal mucus is mostly made of water, which helps give it its slippery, moist texture. In CF, the dysfunction of CFTR means not enough water gets into mucus, so the mucus ends up abnormally thick and sticky.
The porosome is a molecular structure at the surface of cells where tiny vesicles dock to release contents to the outside. CFTR normally interacts with the porosome, and this interaction is thought to play a role in CFTR’s function.
“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,” said Guillermo Marmol, CEO of Porosome Therapeutics.
Porosome Therapeutics’ patent application covers two approaches to treat CF by targeting the porosome. One strategy is to insert a healthy version of the porosome into cells, while the other aims to use small molecules to boost porosome function. In either case, the goal is to increase the functionality of this cellular structure to normalize mucus production.
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.
Preclinical data have indicated that these approaches can help to restore normal mucus production in CF models.
“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,” said Jena, a professor at Wayne State University, in Detroit, who helped to discover the porosome.
Marmol said the company’s new treatment platform for CF and other related disorders comes “following decades of research” by Jena.
“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,” Marmol said.