AMT Receives Orphan Drug Designation from FDA for AMT 011 for the Treatment of Lipoprotein Lipase Deficiency

Amsterdam, The Netherlands – June 4, 2007 – Amsterdam Molecular Therapeutics (AMT), a leader in the field of human gene therapy, today announced that its lead product, AMT 011, has received Orphan Drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of Lipoprotein Lipase (LPL) Deficiency.

In the United States, Orphan Drug designation entitles AMT to exclusive AMT 011 marketing rights for seven years for the treatment of LPL deficiency. The designation also allows AMT to apply for research funding, tax credits for clinical studies, and a waiver from the FDA application user fee required by the Prescription Drug User Fee Act (PDUFA).

"We are pleased to have received this U.S. Orphan Drug designation for AMT 011 in the treatment of LPL deficiency, in addition to the designation we had already secured in Europe," said Ronald Lorijn, Chief Executive Officer of AMT. "These designations are key to our strategic focus on rare diseases and underpin our business model.”

In a study of eight subjects with Complete LPL deficiency presented on May 30, 2007 at the 10th Annual Meeting of the American Society of Gene Therapy, AMT's lead product was shown to be safe and efficacious. Based on the results from the pre-registration Phase I/II trial with AMT-011 the company should have sufficient clinical data by the end of 2007 to file for market authorization in Europe in the first quarter of 2008, followed by filings in the U.S. and Canada.

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About AMT

Amsterdam Molecular Therapeutics (AMT), which has recently announced its intention to seek a listing on Euronext Amsterdam through an IPO, is a leading gene therapy company, with a unique platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy to become a mainstay of clinical medicine. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. As such, AMT’s proprietary platform holds tremendous promise for thousands of rare (orphan) diseases that are caused by one faulty gene. AMT currently has a product pipeline with six products at different stages of development. The company's Management, Supervisory Board and Scientific Advisory Board bring together an extensive know-how of genetics and the biotech and pharmaceutical industries. The company was founded in 1998 by scientists of the University of Amsterdam Medical Center (AMC).

For information:

AMT                                                                  Northbank Communications:
Ronald Lorijn, CEO                                      Adam Michael, Account Director
Phone: +31 20 566 7394                            +44 20 7268 3233
Email: r.h.lorijn@amtbv.com                      amt@northbankcommunications.com