ION859 (formerly IONIS-BIIB7Rx), also known as BIIB094, is an investigational antisense medicine designed to inhibit the production of the leucine-rich repeat kinase 2 (LRRK2) protein as a potential therapy for Parkinson’s disease (PD). The most common genetic mutations in PD are found in the LRRK2 protein. It is believed that increased LRRK2 protein activity could be one of the key drivers for developing PD.
About Parkinson’s Disease
PD is a progressive neurodegenerative disease characterized by loss of neurons in the motor system. Patients with PD can experience tremors, loss of balance and coordination, stiffness, slowing of movement, changes in speech and in some cases cognitive decline. PD is ultimately fatal. There are treatments that can relieve symptoms, but there are no approved disease modifying therapies.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.