Dutch gene therapy firm Amsterdam Molecular Therapeutics BV has obtained an exclusive license to a novel therapeutic agent which is being developed as a treatment for acute intermittent porphyria. The drug, known as AMT-020, was originated in a collaboration between Spanish companies UTE CIMA, Proyecto de Biomedicina CIMA, SL and Digna Biotech, all of which are part of the University of Navarra, in Pamplona, Spain.
AMT explained that the drug is a gene therapy-based product that utilizes adeno-associated viral vector technology for delivery. It is designed to provide a working version of the porphobilinogen deaminase gene to patients who suffer AIP.
The Dutch firm went on to say that the deal follows its successful collaboration with CIMA, which indicated that the agent is safe and has clinical efficacy. Financial terms of the accord were not provided.
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