CAMBRIDGE, Mass.--(BUSINESS WIRE)--Agilis Biotherapeutics, LLC (Agilis), a biotechnology company advancing innovative gene therapies for rare genetic diseases that affect the central nervous system (CNS), announced today that the Company has entered into an exclusive worldwide license agreement with National Taiwan University (NTU) for the treatment of Aromatic L-Amino Acid (AADC) Deficiency using gene therapy developed by Dr. Paul Hwu, Professor of Pediatrics at NTU Hospital. AADC Deficiency is a rare CNS disorder arising from a reduction in the enzyme, aromatic L-amino acid decarboxylase (AADC), caused by mutations in the dopa decarboxylase (DDC) gene. This reduction leads to deficits in the neurotransmitters dopamine, norepinephrine, epinephrine, serotonin and melatonin. In its profound forms, AADC Deficiency causes severe developmental delays, the inability to develop motor strength and control (global muscular hypotonia/dystonia) resulting in breathing, feeding, and swallowing problems, frequent hospitalizations, and the need for life-long care that ultimately culminates in premature death within the first decade of life. In an effort to address the high unmet medical need in AADC patients, Dr. Hwu and colleagues have developed a novel gene therapy targeted to restore deficient AADC and have successfully treated 18 subjects in two prospective clinical studies.
