Pipeline
Disease Indication
Parkinson’s Disease
Parkinson’s disease (PD) is a chronic, degenerative neurological disorder that affects one in 100 people over the age of 60. PD is the second most common neurodegenerative disease, with prevalence more than doubling over the last 2 decades. There is no objective test or biomarker for Parkinson’s disease, so the rate of misdiagnosis can be relatively high. As a result, estimates of the number of people living with the disease vary, although recent research indicates that at least one million people in the United States, and more than five million worldwide, suffer from Parkinson’s disease.
Loss of dopaminergic neurons in the substantia nigra impair basal ganglia circuits which result in characteristic clinical signs of slow movement, tremor and rigidity. Whilst PD is likely to be multifactorial, mitochondrial dysfunction is recognized as a key pathophysiological mechanism in PD. Loss of function mutations in Parkin, PINK1 and protein deglycase DJ-1 predispose to development of early onset PD. Alpha-synuclein, a key potential pathogenic protein in PD, directly interacts with mitochondria at TOM20 and functionally at mitochondrial complex I, inhibiting mitochondrial function. Furthermore, oxidative stress output from mitochondria can oxidize dopamine which further causes alpha-synuclein aggregation and neurotoxicity.