A biopharmaceutical company focused on discovery and development of small-molecule drugs to treat genetic and rare diseases.
In August 2018, PTC announced its acquisition of Agilis BiotherapeuticsAgilis Biotherapeutics, a private biotechnology company focused on the development of gene therapy programs for rare genetic disorders that affect the central nervous system. Through this acquisition, PTC added GT-AADC, an adeno-associated virus (AAV) gene therapy that treats Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency.