Transaction covers technique that allows proteins marked for destruction to escape enzymatic digestion.

Axentis Pharma has obtained patents from BioDevelops Pharma related to a cystic fibrosis treatment. The acquired therapeutic approach enables proteins, which get destroyed in spite of have some functionality, to escape.

In cystic fibrosis, proteins that are designed to regulate the concentration of salt in the epithelial cells of the lungs and other organs have mutations due to a genetic defect. The body thus destroys these mutated proteins, even though they are sufficiently functional.

“In patients with cystic fibrosis,” Gergely Lukacs, M.D., Ph.D., who guided the experimental setup in his labs at the Sick Kids Hospital in Toronto explains, “the protein CFTR (cystic fibrosis transmembrane conductance regulator) with mutated structure is marked with ubiquitin. These ubiquitin-marked proteins are degraded by multiple mechanisms prematurely. If this marker is missing, the protein evades destruction and can regulate the concentration of water and salt secretion in the lung despite its minor structural defect.”

The agreement between Axentis Pharma and  BioDevelops Pharma includes two patent families with a series of international patent applications. They cover the use of molecules for the enzymatic removal of ubiquitin markers in incorrectly folded proteins. The inventions also protect the use of Velcade in cystic fibrosis.

Swiss company Axentis Pharma formed less than a year ago with patent-pending technology to develop drugs for diseases caused by incorrect protein folding in the endoplasmic reticulum, such as cystic fibrosis. “Just six months ago, we reached agreement with aRigen Pharmaceuticals, Japan, on terms and conditions regarding an exclusive license for developing and marketing a patented system for the liposomal encapsulation of an active ingredient designed to combat lung infections in patients with cystic fibrosis,” remarks Joerg Zielasek, president of Axentis Pharma. The company is preparing to initiate a Phase II trial in Germany using the acquired technology, tobramycin liposome formulation, against lung infections in cystic fibrosis patients.

“By acquiring the rights to BioDevelops’ technology platform, Axentis Pharma is both enhancing its development pipeline and offering both patients and investors extensive prospects for the treatment of this disease.”

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