AMT Obtains License to Amgen’s GDNF Gene to Develop Treatment for Parkinson’s Disease
Amsterdam Molecular Therapeutics 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.
“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.”
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.
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Topic world Gene therapy
Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.
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Topic world Gene therapy
Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.