Parkinson’s Disease

Parkinson’s Disease is a motor system disorder that primarily affects people older than 50. It is characterized by tremors in the arms, legs, and face; rigidity in the trunk and limbs; and impaired coordination. The disease is chronic and progressive, but there is wide variation in the rate of progression. It is characterized by loss of the brain cells that produce dopamine; some of the available treatments include dopamine replacement which typically helps with the rigidity and slowness of movement, and other drugs that target tremors, and deep brain stimulation, in which implanted electrodes decrease involuntary movement. Gene therapy approaches to Parkinson’s disease include introduction of neuroprotective genes to preserve the remaining dopaminergic neurons, and converting other neurons into dopaminergic neurons by introducing the genes required to produce dopamine in the brain.

Status

To successfully treat diseases such as Parkinson’s disease, gene therapy needs to target the neurons within the brain. AAV9 is significantly more efficient in delivering genes to the CNS than other serotypes, and can distribute gene expression further with one injection. The proposed therapeutic is in the research stage of development.

Market

As many as one million individuals in the U.S. live with Parkinson’s disease. While approximately four percent of people with Parkinson’s are diagnosed before the age of 50, incidence increases with age. The combined direct and indirect cost of Parkinson’s, including treatment, social security payments and lost income from inability to work, is estimated to be nearly $25 billion per year in the United States alone. Medication costs for an individual with PD average $2,500 a year, and therapeutic surgery can cost up to $100,000 dollars per patient.¹