NEW YORK and GERMANTOWN, Md., Dec. 31, 2013 /PRNewswire/ -- Agilis Biotherapeutics, LLC, a synthetic biology-based company focused on rare genetic diseases, and Intrexon Corporation (NYSE: XON), a leader in synthetic biology, announced today an Exclusive Channel Collaboration (ECC) to develop DNA-based therapeutics for Friedreich's ataxia (FRDA), a rare genetic neurodegenerative disease.
Agilis and Intrexon expect DNA-based therapeutics to provide the ability to target underlying disease mechanisms with precision using tightly-controlled gene therapies for patients with rare inherited diseases such as FRDA. Current FRDA therapies are primarily focused on supportive care and symptom relief. There are no FDA-approved treatment options to address the cause of FRDA.
The technical core of Agilis' novel DNA-based therapeutics will utilize Intrexon's UltraVector® platform and RheoSwitch Therapeutic System® (RTS®) to develop gene therapies and genetically-modified cell therapies for treating FRDA. The ECC's planned approach to target FRDA will employ RTS®, a clinically validated inducible gene switch technology that regulates the expression of therapeutic proteins or bioactive RNA in a dose-dependent fashion.
FRDA is an inherited disease caused by a gene mutation that reduces the expression of frataxin, a protein localized in the "power center" of cells known as the mitochondria, and results in a physically debilitating, life-shortening condition. Progression of the disease causes nervous system damage, problems with movement and early death most often caused by cardiac malfunction. FRDA is the most co
|SOURCE Intrexon Corporation|
Copyright©2012 PR Newswire.
All rights reserved