Agilis Pharmaceuticals

Agilis Pharmaceuticals was a clinical-stage biotechnology company focused on developing innovative gene therapies for rare genetic diseases of the central nervous system (CNS). The company's lead program, GT-AADC, was designed to treat aromatic L-amino acid decarboxylase (AADC) deficiency and has since been approved in several markets under the brand name Upstaza. Agilis utilized adeno-associated virus (AAV) vectors to deliver functional genes directly to the brain, targeting the underlying cause of debilitating neurological disorders. In 2018, the company was acquired by PTC Therapeutics to serve as the foundation for their gene therapy division. Their pipeline also included advanced preclinical programs for Friedreich's ataxia, Angelman syndrome, and Huntington's disease.