Agilis Biotherapeutics and National Taiwan University Enter into Worldwide, Exclusive License Agreement for the Gene Therapy Treatment of AADC Deficiency
Jan 28 16
Agilis Biotherapeutics, LLC announced 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. Under the terms of the agreement with NTU, Agilis will sponsor ongoing research and development of the AADC gene therapy in collaboration with Dr. Hwu, including the conduct of ongoing clinical work on AADC gene therapy and completion of nonclinical testing in advance of the pivotal clinical study, anticipated to begin in 2017. Agilis will also support ongoing manufacturing efforts for the gene therapy.
National Taiwan University Presents at Bit's 5th New Energy Forum-2015, Sep-26-2015 11:35 AM
Sep 22 15
National Taiwan University Presents at Bit's 5th New Energy Forum-2015, Sep-26-2015 11:35 AM. Venue: China. Speakers: Ching-Fuh Lin, Director & Distinguished Professor, Innovative Photonics Advanced Research Center.