PTC Therapeutics announced that it has successfully completed the acquisition of Agilis Biotherapeutics (Cambridge, USA), a private biotechnology company focused on the advancement of innovative gene therapy programs for rare genetic disorders that affect the central nervous system (CNS).
With the acquisition, PTC adds GT-AADC, an adeno-associated virus (AAV) gene therapy that treats Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency. AADC Deficiency is a rare CNS disorder that is caused by mutations in the dopa decarboxylase (DDC) gene. There are no treatments that target the underlying cause of this disorder. The results from prospective clinical studies, indicated that treated subjects were found to exhibit substantial gains on motor function and cognitive scales over multiple years following the single gene therapy treatment.
The acquisition also includes gene therapy programs in development, GT-FA, GT-AS, and GT-RLN, for Friedreich Ataxia, Angelman Syndrome and Cognitive Disorders associated with several neurodevelopmental and neurodegenerative disorders, respectively.
PTC is a US science-led, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. Founded 20 years ago, PTC Therapeutics has successfully launched two rare disorder products and has a global commercial footprint. With a headquarters in New Jersey (USA), PTC also has its international office in Dublin (Ireland).