US synthetic biology companies Agilis Biotherapeutics is focused on designing and engineering first-in-class DNA-based therapeutics to improve and save the lives of patients affected by life-threatening or fatal rare diseases for which there are no or limited treatment options.

Its lead program is focused on developing a DNA-based therapy for patients with Friedreich’s ataxia, a rare genetic neurodegenerative disease that results in a physically debilitating, life-shortening condition. It is developing synthetic 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.

In January 2014 it entered into an exclusive channel collaboration with Intrexon (NYSE: XON) to develop DNA-based therapeutics for Friedreich's ataxia (FRDA), a rare genetic neurodegenerative disease. Through the collaboration with Intrexon, it has access to Intrexon’s enabling technologies, including the UltraVector operating system and RheoSwitch Therapeutic System for developing novel DNA-based therapeutics.