Generation 2+ antisense drug
ION859 (formerly IONIS-BIIB7Rx), also known as BIIB094, is an antisense oligonucleotide (ASO) targeting Leucine Rich Repeat Kinase 2 (LRRK2) messenger ribonucleic acid (mRNA). ION859 is designed to prevent the production of LRRK2 protein and is being developed as a potential therapy for Parkinson’s disease (PD). The most common genetic mutations in PD cases are found in the LRRK2 gene. Increased LRRK2 protein activity is hypothesized to be one of the key drivers of PD pathogenesis. It is hypothesized that reduction of LRRK2 mRNA and, subsequently, reduced synthesis of LRRK2 protein will ameliorate the toxic effects of gain-of-function mutations as well as the primary pathology in PD patients without the LRRK2 mutation.
About Parkinson’s Disease
Parkinson’s disease is a progressive neurodegenerative disease characterized by loss of neurons in the motor system. Patient’s with Parkinson’s disease can experience tremors, loss of balance and coordination, stiffness, slowing of movement, changes in speech and in some cases cognitive decline. Parkinson’s disease is ultimately fatal. There are treatments that can relieve symptoms, but there is no disease modifying therapy. The exact cause is unknown, but it is believed to be a combination of genetics and environmental factors. There are known hereditary mutations that cause Parkinson’s disease, including dominantly inherited gain-of-function mutations in the LRRK2 gene.