The goal of the ECC is to develop DNA-based therapeutics to repair or replace the "broken" gene in FRDA and enable increased production of the frataxin protein to alleviate the downstream effects of frataxin deficiency. The combination of gene correction with additional therapeutic modulators in a multigenic approach has the potential to create a potent treatment. This treatment is expected to further improve cardiovascular and neurological function by addressing the underlying causes of FRDA and associated symptoms. Agilis also has an option to advance a second undisclosed rare disease indication.
"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 Biotherapeutics. "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."
Samuel Broder, M.D., Senior Vice President of Intrexon's Health Sector and former Director of the National Cancer Institute, said, "FRDA causes heart failure and progressive neurological deterioration, which in turn cause suffering and premature deaths. The goal of this collaboration is to harness Intrexon's proprietary technologies in synthetic DNA, as well as our expertise in molecular, protein, and cellular engineering, to benefit patients with this very serious disorder."
Intrexon Founder and Chief Science Officer Thomas Reed, Ph.D., remarked that unlike some biotechnology companies which are limited to single classes of t
|SOURCE Intrexon Corporation|
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