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 common hereditary ataxia with an estimated 5,000 to 10,000 patients in the United States.
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 harnessIntrexon'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 therapeutic molecules or gene delivery systems, Intrexon is equipped to pursue several different therapeutic approaches for treating the complexities associated with FRDA.
"As an inventor and integrator of technology platforms, we will make use of our UltraVector® multi-gene engineering, RTS® gene switch, advanced Protein Engineering, and other platforms to develop therapeutic candidates designed to treat both the neurological and the cardiovascular pathologies of FRDA. We are confident that our ability to pursue multiple approaches for treating this complex disease will significantly increase our probability of success.