Cystic Fibrosis Rescued by Reprogramming the Cell’s Secretory Machinery

Porosome Therapeutics, Inc. (Porosome Therapeutics) today announced the publishing of a study that brings promise for a cure to cystic fibrosis (CF). The preprint highlights the ground-breaking therapeutic approach that overcomes CF nearly four times more effectively than currently available medications to treat the inherited disorder.

“We’re excited about the potential the porosome platform holds for developing new and novel drugs and therapies to address a wide range of secretory disorders,” said Professor Bhanu P. Jena, PhD, Founder and Chairman, Porosome Therapeutics. “Echoing the International Patent Search Agency, our central claims are ‘novel’ and ‘inventive,’” said Guillermo Marmol, President and CEO, Porosome Therapeutics. “We look forward to bringing such new, novel, safe, and curative treatments to market for people living with cystic fibrosis, diabetes, and other secretory disorders.”

On December 9, 2023, the preprint entitled “Reprogramming the Cells Secretory Machinery: A Cystic Fibrosis Rescue” was published on bioRxiv. The study identified an innovative therapeutic solution for CF patients by first determining the relationship between the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene and porosome-mediated secretion in human airways. Results demonstrate that mutation in porosome-associated CFTR also affects other proteins within the porosome secretory machine, thereby reducing mucus secretion. Researchers then reconstituted functional porosomes into the plasma membrane of CFTR mutant cells to demonstrate that porosome reconstitution normalizes mucus secretion approximately four times more effectively than currently available CF drugs.

“Our company research has aided in the ability to identify the critical role CFTR plays within the porosome secretory machinery. By harnessing this technology, we can advance innovative treatments for currently incurable diseases,” said Marmol. “Our patient-centered approach in drug design and development at Porosome Therapeutics will transform therapeutic options for patients diagnosed with cystic fibrosis, diabetes, cancer, Alzheimer’s, and other neurological, digestive, and immune disorders.”

CF is a genetic condition that adversely affects cells in the body that secrete mucus, such as the airways, digestive system, and reproductive organs. Normally, these fluids are slick and thin. However, mutations in the CFTR gene in people with CF make the secretions thick and sticky. The secretions clog tubes, ducts, and passages rather than lubricating them, preventing normal function. While more than 2,000 mutations in the CFTR gene have been identified, the ΔF508 CFTR is the most common, accounting for approximately 70% of all CFTR mutations. It is estimated that more than 160,000 people worldwide are living with CF, with a consequent reduction in life expectancy.

With this study, Porosome Therapeutics has demonstrated positive pre-clinical results of porosome-reconstitution therapy in human bronchial air-liquid interface 3D cultures that mimic normal lung physiology, responding to CFTR inhibitors and the CF corrector and modulator drugs tezacaftor and ivacaftor. The company is currently conducting in vivo animal studies.

About Porosome Therapeutics, Inc.

Porosome Therapeutics, Inc. is a Boston-based biopharmaceutical company utilizing the breakthrough discovery of the porosome, the cell’s secretory machinery, and novel technologies to identify and create proprietary high-value therapeutics for currently undruggable porosome proteins relating to secretory and hydration disorders such as cystic fibrosis, diabetes, and cancer.

Porosome Therapeutics is the first company with a technology platform that develops novel therapies targeting the porosome, the universal secretory machinery in cells. This platform is based on decades of research supported by more than 200 publications and is effective against many serious diseases involving secretory defects. The platform also specializes in highly specific nanobody-mediated targeted therapies that greatly reduce drug side effects.

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