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AMT Obtains License to Amgen's GDNF Gene to Develop Treatment for Parkinson's Disease with AMT's Proprietary Gene Therapy Platform

AMSTERDAM, The Netherlands, September 18 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it obtained a license from Amgen to use their GDNF gene for the development of a gene therapy treatment for Parkinson's disease. The combination of this gene with AMT's proprietary adeno-associated virus (AAV) gene therapy platform could potentially allow the development of an effective, long-term treatment for this progressive and crippling disease.

Parkinson's disease is the second most common neurodegenerative disease. It usually affects people over 65 with an estimated total of 4.5 million patients worldwide. Due to increasing life expectancy of the general population, the number of patients with Parkinson's disease is expected to double to around 9 million patients between now and the year 2030.

Patients with Parkinson's disease slowly lose control of their muscles, resulting in tremors, stiffness, slowness of movement, and lack of coordination and thus in a serious loss of quality of life. Parkinson's is caused by degeneration and death of nerve cells in a specific part of the brain. These cells produce dopamine, a substance necessary for communication between nerve cells involved in the coordination of movement. Current therapies are limited to treatment of symptoms. There are no therapies available that slow down or halt the progression of the disease.

A new way to deliver the GDNF gene

"This license from Amgen offers us a unique opportunity to combine our gene technology and know-how with the GDNF gene as a tool to create a potential breakthrough in the treatment for this common and severely debilitating disease," said Ronald Lorijn, CEO of AMT "We believe our gene therapy approach could be an effective way to deliver the gene to the regions of the brain affected by Parkinson's disease."

Protect and improve nerve cells with GDNF

The GDNF gene contains the information for a protein necessary for the development and survival of nerve cells. AMT will combine this gene with its own proprietary technology to develop a gene therapy treatment that aims to protect and enhance the function of the nerve cells that produce dopamine. The positive effect of GDNF on nerve cells has been shown in several animal studies, making it an attractive candidate for the treatment of Parkinson's disease.. AMT believes that its gene-delivery platform may potentially provide a solution for delivering GDNF to the brain.

About Amsterdam Molecular Therapeutics

AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming 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, especially the ones that are caused by one faulty gene. AMT currently has a product pipeline with seven products at different stages of development.

About Amgen

Amgen discovers, develops, manufactures and delivers innovative human therapeutics. A biotechnology pioneer since 1980, Amgen was one of the first companies to realize the new science's promise by bringing safe and effective medicines from lab, to manufacturing plant, to patient. Amgen therapeutics have changed the practice of medicine, helping millions of people around the world in the fight against cancer, kidney disease, rheumatoid arthritis, and other serious illnesses. With a deep and broad pipeline of potential new medicines, Amgen remains committed to advancing science to dramatically improve people's lives. To learn more about our pioneering science and our vital medicines, visit

Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and expectations for future operations, prospects and financial condition. Words such as "strategy," "expects," "plans," "anticipates," "believes," "will," "continues," "estimates," "intends," "projects," "goals," "targets" and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of Amsterdam Molecular Therapeutics only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of AMT. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting AMT's business, including, but not limited to, the timely commencement and success of AMT's clinical trials and research endeavors, delays in receiving U.S. Food and Drug Administration or other regulatory approvals (i.e. EMEA, Health Canada), market acceptance of AMT's products, effectiveness of AMT's marketing and sales efforts, development of competing therapies and/or technologies, the terms of any future strategic alliances, the need for additional capital, the inability to obtain, or meet, conditions imposed for required governmental and regulatory approvals and consents. AMT expressly disclaims any intent or obligation to update these forward-looking statements except as required by law. For a more detailed description of the risk factors and uncertainties affecting AMT, refer to the prospectus of AMT's initial public offering on June 20, 2007, and AMT's public announcements made from time to time.

SOURCE Amsterdam Molecular Therapeutics B.V
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