Cystic Fibrosis - Axentis Pharma Takes Over Patents and Continues Development of Innovative Platform Technology

Friday, June 27, 2008 General News
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ZURICH, Switzerland, June 26 The rights to key patentsfor an innovative screening tool for treatments for cystic fibrosis weretransferred today to Swiss biotech company Axentis Pharma AG. The company hasacquired the rights from an Austrian firm that has made a vital contributionto the development of new approaches to treating the disease. The coreelement of the platform technology involves human proteins which - despiteoffering sufficient functionality - are subjected to enzymatic digestion inuntreated cells due to genetic mutations in their structure.

Axentis Pharma AG (Switzerland) today announced that, as part of itstakeover of an Austrian research company, it has also acquired two patentfamilies that incorporate a series of international patent applications. Thecompany is taking over these rights from Austrian-based BioDevelops PharmaGmbH, which three years ago identified the therapeutic potential offered by anew approach to the treatment of cystic fibrosis and secured the relevantrights.

The technology directly targets the functional cause of cystic fibrosis -the process whereby the body destroys mutated proteins which were originallydesigned to regulate the concentration of salt in the epithelial cells of thelungs and other organs. In patients with cystic fibrosis, a genetic defectmeans that many such proteins are produced with minor structural mutations.Although the mutated proteins still offer sufficient functionality, they areeliminated by quality control functions in the endoplasmic reticulum of thecells. The therapeutic approach now under development at Axentis Pharmaenables these proteins - which are still functional despite their mutatedstructures - to escape enzymatic digestion.

Dr. Gergely Lukacs who guided the experimental set-up in his labs at theSick Kids Hospital in Toronto explains: "In patients with cystic fibrosis,the protein CFTR (cystic fibrosis conductance regulator) with mutatedstructure is marked with ubiquitin. These ubiquitin-marked proteins aredegraded by multiple mechanisms prematurely. If this marker is missing, theprotein evades destruction and can regulate the concentration of water andsalt secretion in the lung despite its minor structural defect.

Joerg Zielasek, president of Axentis Pharma AG, comments on what theacquisition of these rights means for the company: "Just six months ago, wereached agreement with aRigen Pharmaceuticals, Japan, on terms and conditionsregarding an exclusive license for developing and marketing a patented systemfor the liposomal encapsulation of an active ingredient designed to combatlung infections in patients with cystic fibrosis. By acquiring the rights toBioDevelops' technology platform, Axentis Pharma is both enhancing itsdevelopment pipeline and offering both patients and investors extensiveprospects for the treatment of this disease."

About cystic fibrosis

Cystic fibrosis is the most common life-threatening hereditary diseaseamong Caucasian population. The disease is caused by a mutation in the CFTR(Cystic Fibrosis Transmembrane Conductance Regulator) gene found onchromosome 7. This mutation causes increased secretion deposits on mucousmembranes. Lung complications represent the most serious manifestation of thedisease - and the reason for the high mortality rate amongst patients. Suchcomplications often involve infection of the bronchi by the bacteriaPseudomonas aeruginosa. Chronic inflammations then cause lung functions tobecome blocked. Besides the break-down of lung tissue, this also leads tobronchiectasis and lung failure.

About Axentis Pharma AG (

Axentis Pharma AG is a Swiss biotechnology company. The company is usinga patent-pending platform technology to develop therapies for diseases causedby incorrect protein folding in the endoplasmic reticulum. The most prominentexample of such diseases is

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