AMSTERDAM, The Netherlands, September 18 Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field ofhuman gene therapy, today announced that it obtained a license from Amgen touse their GDNF gene for the development of a gene therapy treatment forParkinson's disease. The combination of this gene with AMT's proprietaryadeno-associated virus (AAV) gene therapy platform could potentially allowthe development of an effective, long-term treatment for this progressive andcrippling disease.
Parkinson's disease is the second most common neurodegenerative disease.It usually affects people over 65 with an estimated total of 4.5 millionpatients worldwide. Due to increasing life expectancy of the generalpopulation, the number of patients with Parkinson's disease is expected todouble 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 ofcoordination and thus in a serious loss of quality of life. Parkinson's iscaused by degeneration and death of nerve cells in a specific part of thebrain. These cells produce dopamine, a substance necessary for communicationbetween nerve cells involved in the coordination of movement. Currenttherapies are limited to treatment of symptoms. There are no therapiesavailable 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 ourgene technology and know-how with the GDNF gene as a tool to create apotential breakthrough in the treatment for this common and severelydebilitating disease," said Ronald Lorijn, CEO of AMT "We believe our genetherapy approach could be an effective way to deliver the gene to the regionsof 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 thedevelopment and survival of nerve cells. AMT will combine this gene with itsown proprietary technology to develop a gene therapy treatment that aims toprotect and enhance the function of the nerve cells that produce dopamine.The positive effect of GDNF on nerve cells has been shown in several animalstudies, making it an attractive candidate for the treatment of Parkinson'sdisease.. AMT believes that its gene-delivery platform may potentiallyprovide a solution for delivering GDNF to the brain.
About Amsterdam Molecular Therapeutics
AMT has a unique gene therapy platform that to date appears to circumventmany if not all of the obstacles that have prevented gene therapy frombecoming a mainstay of clinical medicine. Using adeno-associated viral (AAV)vectors as the delivery vehicle of choice for therapeutic genes, the companyhas been able to design and validate what is probably the first stable andscalable AAV production platform. As such, AMT's proprietary platform holdstremendous promise for thousands of rare (orphan) diseases, especially theones that are caused by one faulty gene. AMT currently has a product pipelinewith seven products at different stages of development.
Amgen discovers, develops, manufactures and delivers innovative humantherapeutics. A biotechnology pioneer since 1980, Amgen was one of the firstcompanies to realize the new science's promise by bringing safe and effectivemedicines from lab, to manufacturing plant, to patient. Amgen therapeuticshave changed the practice of medicine, helping millions of people around theworld in the fight against cancer, kidney disease, rheumatoid arthritis, andother serious illnesses. With a deep and broad pipeline of potential newmedicines, Amgen remains committed to advancing science to dramaticallyimprove people's lives. To learn more about our pioneering science and ourvital medicines