Under this collaboration, the companies will seek to develop therapeutics to repair or replace the deficient gene that leads to Friedreich’s ataxia. In addition, Agilis will have an option to advance a second, undisclosed rare disease indication as well. Agilis will make use of Intrexon’s UltraVector platform and RheoSwitch Therapeutic System to develop gene therapies and genetically modified cell therapies for the treatment of Friedreich’s ataxia. The RheoSwitch Therapeutic System is a clinically validated inducible gene switch technology capable of regulating expression of therapeutic proteins or bioactive RNA in a dose-dependent manner. No specific terms or financial details for the agreement were disclosed.

 

“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,” George S. Zorich, CEO of Agilis, said in a press release about the collaboration. “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.”

 

Friedreich’s ataxia is a genetic disease that develops as a result of a gene mutation that reduces expression of the protein frataxin, which is found in the mitochondria of cells. as the disease progresses, it causes damage to the nervous system, problems with movement and early death, which is most often the result of cardiac malfunction. Friedreich’s ataxia is the most common hereditary ataxia, and some 5,000 to 10,000 patients suffer from this disease in the United States. Currently, existing therapies for the disease consist of supportive care and symptom relief only, and no U.S. Food and Drug Administration-approved therapies exist that address the underlying cause of Friedreich’s ataxia.

 

“[Friedreich’s ataxia] 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,” Samuel Broder, M.D., senior vice president of Intrexon’s Health Sector and former director of the National Cancer Institute, commented.

 

Intrexon and Agilis will seek to develop therapeutics capable of repairing or replacing the defunct gene and increasing producing of the frataxin protein, a treatment that, it is believed, will improve cardiovascular and neurological function by targeting the underlying cause of the disease.

 

“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,” Thomas Reed, Ph.D., founder and chief science officer of Intrexon, noted in a statement. “We are confident that our ability to pursue multiple approaches for treating this complex disease will significantly increase our probability of success.”

 

 

SOURCE: Intrexon press release