AMT will evaluate delivery of this gene using its adeno-associated virus technology.
Amsterdam Molecular Therapeutics (AMT) licensed Amgen’s GDNF gene for use as a gene therapy for Parkinson’s disease. AMT will use its adeno-associated virus (AAV) technology in the assessment the gene’s potential to protect and enhance the function of the nerve cells that produce dopamine.
The GDNF gene contains the information for a protein necessary for the development and survival of nerve cells. The positive effect of GDNF on nerve cells has been shown in several animal studies, according to AMT.
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.