PTC Therapeutics has successfully completed the acquisition of Agilis Biotherapeutics, Inc., a private biotechnology company focused on the advancement of innovative gene therapy programs for rare genetic disorders that affect the central nervous system (CNS). 

“We are excited to complete the acquisition and welcome the Agilis employees to PTC,” said Stuart W. Peltz, Ph.D., chief executive officer, PTC Therapeutics, Inc. “We have added a team with exceptional gene therapy experience, a targeted micro-delivery gene therapy platform that has demonstrated durable clinical benefit, as well as gene therapy manufacturing and R&D capabilities. This acquisition deepens our pipeline and, as a combined organization, we will be able to optimally treat more rare disorders and accelerate the process of bringing important new therapies to patients worldwide.”

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.