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Last Updated Monday, April 21 2014

Agilis, Intrexon in Rare Disease Pact

By Kristin Brooks

Published January 2, 2014
Agilis Biotherapeutics and Intrexon Corp. have entered an Exclusive Channel Collaboration (ECC) to develop DNA-based therapeutics for Friedreich's ataxia (FRDA), a rare genetic neurodegenerative disease. The companies will explore DNA-based therapeutics to directly target underlying disease mechanisms using tightly-controlled gene therapies for patients with rare inherited diseases such as FRDA.
Agilis will employ its Intrexon's UltraVector platform and RheoSwitch Therapeutic System (RTS) to develop gene therapies and genetically-modified cell therapies for treating FRDA. The collaboration aims to target FRDA employing RTS, a clinically validated inducible gene switch technology to repair or replace the "broken" gene in FRDA and allow increased production of the frataxin protein to alleviate the effects of frataxin deficiency.
Current FRDA therapies are primarily focused on symptom relief and there are no FDA-approved drugs to treat the cause of FRDA. FRDA is an inherited disease that results in nervous system damage, problems with movement and early death most often caused by cardiac malfunction. FRDA affects an estimated 5,000 to 10,000 patients in the U.S.   
"We are thrilled to be working with Intrexon with the hope of providing children and adults affected with rare genetic disorders with promising new treatments," said George S. Zorich, chief executive officer of Agilis. "We believe Agilis is on the forefront of one of the most promising treatment breakthroughs for Friedreich's ataxia. I am personally excited to collaborate with the Intrexon team and look forward to developing new transformative therapies together."

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