Amsterdam Molecular Therapeutics BV receives global rights to develop and commercialize AMT-020 for acute intermittent porphyria
Rights from University of Navarra, Pamplona, Spain strengthen AMT's portfolio of gene therapy products
Amsterdam Molecular Therapeutics BV (AMT) announced that it has obtained exclusive worldwide rights to develop and commercialise AMT-020 as a therapeutic product from UTE CIMA, Proyecto de Biomedicina CIMA, S.L. and Digna Biotech (all parties part of the University of Navarra, Pamplona, Spain).
The licence allows AMT to initiate the clinical development of AMT-020 to treat acute intermittent porphyria. This follows successful collaboration between CIMA and AMT, demonstrating the safety and preclinical efficacy of the product, AMT-020. 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.
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Topic world Gene therapy
Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.
Topic world Gene therapy
Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.