Agilis and Intrexon team up to tackle Friedreich’s Ataxia

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Agilis Biotherapeutics, a biotech focused on the design and creation of cutting edge DNA-based therapeutics, have announced an exclusive channel collaboration with Intrexon Corporation to develop theraputics designed to treat the cause of Friedreich’s Ataxia.

This is the latest in a sting of collaborations made by Intrexon, a synthetic biology company focused on working with bitechnology firms to deliver life improving advances in biology through the use of it’s ‘Better DNA’ approach.

The collaboration will give Agilis access to Intrexon’s UltraVector Platform for DNA construction and their RheoSwitch Therpeutic Systems which allows for tightly regulated dose-dependant expression. With these technologies Agilis seeks to develop novel DNA-based therapeutic treatments which can target the underlying cause of Freidreich’s Ataxia.

Freidreich’s Ataxia is an inherited life-shortening and physically debilitating disease affecting nervous system. With an estimated 5,000-10,000 patients in the US alone, freideich’s Ataxia is the most common heritary form of atxia. But as of yet, the only FDA-apporved treatments look to treat the symptoms, not the cause, of the disease. The cause of Freidreich’s ataxia is a mutation in the FXN which causes a excess production of the protein frataxin. This excess then begins to disrupt the normal workings of mitochondria which can result in cell damage leading to movement problems and worsening muscle coordination (ataxia)

Agilis’ and Intrexon hope that they can produce a treatment capable of replacing or ‘fixing’ the mutated gene. Through a mixture of gene correction and additional therapeutic modulators, the goal of the treatment is to improve patient’s cardiovascular and neurological functions.

“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… 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.”
George S. Zorich, Chief Executive Officer of Agilis Biotherapeutics

See the press release here.

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