PTC Therapeutics completes acquisition of Agilis Biotherapeutics
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. ■