Amsterdam Molecular Therapeutics (AMT) obtained exclusive, worldwide rights to develop and commercialize AMT-020 as a therapeutic product from UTE CIMA, Proyecto de Biomedicina CIMA, and Digna Biotech (all part of the University of Navarra, Pamplona, Spain).
The license allows AMT to initiate the clinical development of AMT-020 to treat acute intermittent porphyria. AMT-020 is an AAV vector gene therapy containing the porphobilinogen deaminase gene, which encodes for the enzyme that is defective in acute intermittent porphyria. This disease is associated with recurrent attacks of abdominal pain, gastrointestinal dysfunction, and neurologic disturbances.