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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

product will be to confirm the ability of our liver-directed vector technology to induce long-term therapeutic protein expression in non-human primates."

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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 www.amtbv.com <http://www.amtbv.com/> .

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.

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