Agilis Biotherapeutics and Intrexon collaborate on rare genetic disease

US synthetic biology companies Agilis Biotherapeutics and Intrexon (NYSE: XON) have entered an exclusive channel collaboration (ECC) to develop DNA-based therapeutics for Friedreich's ataxia (FRDA), a rare genetic neurodegenerative disease.

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 Therapeutics 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.