PTC Therapeutics Inc. acquires Agilis Biotherapeutics and with it GT AADC a treatment for Aromatic L-Amino Acid Decarboxylase Deficiency.

PTC Therapeutics, Inc. announced that it has entered into an agreement to acquire Agilis Biotherapeutics, Inc., a biotechnology company advancing an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system (CNS). The transaction was approved by the Boards of both companies. The lead gene therapy candidate, GT-AADC, has compelling clinical data in treating Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency. AADC Deficiency is a rare CNS disorder arising from reductions in the enzyme AADC that result from mutations in the dopa decarboxylase (DDC) gene. In its severe forms, AADC Deficiency causes the inability to develop any motor control (global muscular hypotonia/dystonia) resulting in breathing, feeding, and swallowing problems, frequent hospitalizations, and the need for life-long care. Many patients die in the first decade of life due to profound motor dysfunction and secondary complications such as choking, hypoxia, and pneumonia. Currently, no treatment options are available for the underlying cause of the disorder, and care is limited to palliative options with significant burden on caregivers.