human gene therapy, 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 amtbv.
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.
Amsterdam Molecular Therapeutics B.V
amtbv
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