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Amsterdam Molecular Therapeutics BV to present preclinical,proof-of-concept data on its gene therapy AMT-020 to treat acute,intermittent porphyria

The research, done in collaboration with the Centro de Investigación Médica Aplicada (CIMA) at the University of Navarra, Pamplona, Spain, is being presented at Porphyrins and Porphyrias Congress, Rotterdam, the Netherlands.

AMSTERDAM, the Netherlands, May 1st 2007 - Amsterdam Molecular Therapeutics BV (AMT), a leader in the field of human gene therapy, will today present preclinical data on its AMT-020 gene therapy to treat acute intermittent porphyria (AIP) at the Porphyrins and Porphyrias Congress, Rotterdam.

The data suggest that AMT-020 has the potential to protect patients suffering from recurrent porphyria attacks and/or neuropathy.

AMT-020 is comprised of an Adeno-Associated Virus (AAV) vector delivering the gene for porphobilinogen deaminase (PBGD). The aim of this study, which was performed by Dr's Fontanellas, González-Aseguinolaza and Prieto at CIMA, was to investigate the potential of AMT-020 to protect against acute attack induced by phenobarbital (Pb) in a mouse model of AIP. The model reproduces key features of AIP attack after Pb induction, including increased urinary excretion of haem precursors and decreased motor function.

PBGD-deficient female and male mice were intravenously injected with 1x1011 gc/mouse of AMT-020. Control animals received the same AAV vector but carried the luciferase reporter gene.

One month after injection, both groups were treated with an increasing dose of Pb for 4 days to induce a porphyria attack. The results showed that porphyric mice that received AMT-020 had almost normal haem precursors in the urine and normal motor function, indicating that the treatment completely prevented the occurrence of porphyric attacks.

"These results are very encouraging, and show the potential of AMT-020 to treat AIP," said Sander van Deventer, CSO at AMT, who presented the data. "The next stage in the development of the product will be to confirm the ability of our liver-directed vector technology to induce long-term therapeutic protein expression in non-human primates."


About AMT

Amsterdam Molecular Therapeutics BV (AMT) is a gene therapy company founded by scientists of the University of Amsterdam Medical Center (AMC) in 1998. AMT focuses on the development of gene-based therapies for orphan metabolic and ocular diseases. AMT's long-term gene expression technology is based on specific delivery of therapeutic genes into target organs or tissues. Production of AAV-based gene therapy vectors has been optimized and AMT has developed and validated a unique, stable and scalable GMP production platform. Its lead product, AMT-011, is in phase II for the first indication: treatment of lipoprotein lipase deficiency type I.

The company's Management, Supervisory and Scientific Advisory Board bring together an extensive know-how from both gene science as well as the biotech and pharmaceutical worlds. For further information, go to <> .

About Acute Intermittent Porphyria

Acute intermittent porphyria is a life threatening inherited disease characterised by a partial deficiency of hepatic porphobilinogen deaminase (PBGD). Clinical symptoms include attacks of acute abdominal pain, muscular weakness and a complex array of neurovisceral and psychiatric symptoms. There is also an increased risk of primary liver cancer and renal failure. The attacks are normally triggered when the haem synthesis is induced by infections, stress, alcohol, hormones, or specific drugs. The prevalence of symptomatic disease is 1-2 per 20,000 individuals, however, in some regions of Sweden the prevalence of the disease can be as high as 60-100 cases per 100,000 population. Women are more commonly affected than men.

About the Cent ro de Investigación Médica Aplicada at the University of Navarra

CIMA is a biomedical research project which aims to improve clinical practice. It was started at the University of Navarra in order to give an organized and systematic response to the need to fight against severe diseases that currently cannot be cured.

About the Porphyrins and Porphyrias Congress 2007

The Porphyrins & Porphyrias meetings are biennial events organised with the aim of providing an environment for active participation of both experienced and new researchers in the field. This years event is held in Rotterdam, The Netherlands between April 29th and May 3rd.

There will be presentations and discussions on all haem synthesis enzymes, including structure and function, molecular/genetic aspects and clinical syndromes. Haem degradation (including haem oxygenase, bilirubinaemias etc.) and iron related topics, and general porphyria. Diagnostics/therapies/clinical issues, including drug porphyrinogenicity will also be covered.

For further information, please contact:


Prof. Sander Van Deventer, CSO

Phone: +31 20 566 7394


Northbank Communications:

Adam Michael, Account Director

Phone: +44 20 7268 3233


Best regards

Holly Griffiths Account Executive --------------------------------------------- Direct line: +44 (0)20 7268 3241 Main tel: +44 (0)20 7268 3002 Fax: +44 (0)20 7268 3102 <>

Northbank Communications Ltd 85 Tottenham Court Road, London, W1T 4TQ, UK


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