Agilis Biotherapeutics

Agilis Biotherapeutics was a clinical-stage biotechnology company dedicated to developing innovative DNA therapeutics for rare genetic diseases of the central nervous system. The company specialized in using adeno-associated virus (AAV) vectors to deliver functional genes directly to target tissues, with a primary focus on neurological disorders. Their lead program, GT-AADC, was developed for the treatment of Aromatic L-amino acid decarboxylase (AADC) deficiency, a devastating orphan disease. In 2018, Agilis was acquired by PTC Therapeutics, which integrated the Agilis pipeline to bolster its gene therapy capabilities. The lead asset has since received regulatory approval under the brand name Upstaza.