About Parkinson’s disease
Currently, more than 10 million people worldwide are estimated to be living with Parkinson’s disease, making it the world’s second most prevalent neurodegenerative disease, and most frequent movement disorder. According to the Parkinson’s Foundation, nearly 90,000 people are diagnosed with Parkinson’s disease in the U.S. each year. As many as 90% of Parkinson’s disease cases have no known specific cause, while the remainder show genetic inheritance. The average age for onset of Parkinson’s is with adults in their late fifites, and the incidence increases with age.
Distinguishing features of Parkinson’s disease include resting tremor, stiffness, slowness of movement and postural instability. There are a variety of clinical features that also commonly occur prior to those, including but not limited to sleep disturbances, constipation and behavioral abnormalities.
Parkinson’s disease is an age-related degenerative brain condition, meaning it causes parts of your brain to deteriorate over time. Deep in down in the brain, there is an area called the substantia nigra where cells produce dopamine, a chemical that carries messages around your brain to your nerve cells that control body movements, from walking and talking to eating and going to the bathroom. Some with Parkinson’s may not even realize they have the disease until as many as 80% of their dopamine producing cells are no longer working, thus symptoms present themselves differently for every individual.
Clinical Trials
We are enrolling patients with moderate stages of Parkinson’s disease to assess the safety and preliminary efficacy of GDNF gene therapy. Parkinson’s is the fastest growing neurological condition in the world. There is currently no cure for this progressive movement disorder, but AskBio is committed to investigating a possible option to address unmet needs in Parkinson’s disease.
Glial cell line-derived neurotrophic factor (GDNF) gene therapy for Parkinson’s disease
AB-1005 is currently in trials with the therapeutic intent to encourage appropriate levels of naturally occurring cell growth that promotes the survival and functioning of vulnerable brain cells that degenerate in Parkinson’s disease. GDNF gene therapy is designed to take advantage of the brain’s own cellular production, to encourage natural growth of dopamine cells with a goal to restore continuous production of GDNF protein, necessary for the recipient nerve cells to trigger healthy body movement and various functions.
AB-1005 is delivered directly to the brain via a special operation that uses real-time MRI to allow the surgeon to accurately deliver the therapeutic payload.