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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
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