Cystic fibrosis (CF), a disease caused by a mutation in the gene known as CFTR (Cystic Fibrosis Transmembrane Conductance Regulator), affects the secretion deposits on mucous membranes in the respiratory and digestive system. In the lungs, some of the most common complications involve infection of the bronchi, chronic inflammations, bronchiectasis and lung failure.
CF complications are generally caused by a malfunction in the endoplasmic reticulum, which leads to proteins with incorrect conformations. Currently, the Swiss biotech company Aventis Pharma AG is developing therapies for diseases caused by incorrect function in the endoplasmic reticulum (as is the case of cystic fibrosis) using a patent-pending platform technology.
In order for Aventix to continue developing a successful screening tool for treatment for CF, the company has acquired the rights to key patents from an Australian firm, BioDevelops Pharma GmbH, including two patent sets that incorporate diverse international patent applications. The patented technology involves detection human proteins exposed to enzymatic digestion in protected cells, due to the alterations in their structure.
In patients with cystic fibrosis, the protein that regulates the concentration of salt in epithelial cells is constructed with structural alterations that are specific to those with the disease. Even when these proteins can offer satisfactory functionality, they are eliminated by the endoplasmic reticulum of the cells. The therapeutic methodology in development at Axentis Pharma enables these proteins to outflow enzymatic digestion
The president of Axentis Pharma AG, Joerg Zielasek, recently explained the importance of obtaining these rights for the company: “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.
Because the acquired rights have already been approved for the use of the proteasome inhibitor Bortezomib for second medical use in humans, the company is now in the final preparations to initiate a phase II clinical trial using a formulation of tobramycin liposome against lung infections in patients with cystic fibrosis. 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.”