The combination of this gene with Amsterdam Molecular Therapeutics’s (AMT) proprietary adeno-associated virus gene therapy platform could potentially allow the development of an effective, long-term treatment for this progressive and crippling disease.
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.
Ronald Lorijn, CEO of AMT, said: “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. 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.”