Parkinson’s disease is a progressive neurodegenerative disorder affecting nearly one million Americans and approximately 10 million people worldwide.
Parkinson’s is caused by degeneration of dopaminergic neurons which affect motor control and function. In addition to cardinal symptoms such as shaking or tremors, bradykinesia (slow movement), and rigidity, dopamine loss also commonly leads to additional non-motor symptoms, including cognitive impairment, mood, and behavioral changes.
Parkinson’s is typically first diagnosed after age 60. Only ~10% of patients experience first symptoms before the age of 50. Approximately 60,000 new cases are diagnosed each year in the U.S. alone.
Current treatments provide symptomatic relief, but none modify the underlying progression of the disease. Around 50% of Parkinson’s patients stop responding adequately to oral dopaminergic therapies within five years of diagnosis, leading to significant deterioration of motor symptoms and a poor quality of life. Deep brain stimulation (DBS) is the established standard of care for patients no longer responding to oral therapies, however, while effective in controlling motor symptom, the treatment is invasive and carries safety concerns.
We believe gene therapy has the potential to be a safer, less burdensome, and superior treatment option than what is currently available to patients. Learn more about our investigational gene therapy AAV-GAD, a novel, one-time treatment that we believe offers a simpler and safer alternative to current standards of care.