AMT Obtains License to Amgen's GDNF Gene to Develop Treatment for Parkinson's Disease
News Sep 19, 2008
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.
In a new study in cells, University of Illinois researchers have adapted CRISPR gene-editing technology to cause the cell’s internal machinery to skip over a small portion of a gene when transcribing it into a template for protein building. This gives researchers a way not only to eliminate a mutated gene sequence, but to influence how the gene is expressed and regulated.