Chemistry: Generation 2+
ION464 (formerly IONIS-BIIB6Rx), also known as BIIB101, is an investigational antisense medicine targeting alpha-synuclein (SNCA) messenger ribonucleic acid (mRNA). ION464 is designed to prevent the production of alpha-synuclein protein and is being developed as a potential therapy for Parkinson’s disease (PD), Multiple System Atrophy (MSA) and related synucleinopathies. Alpha-synuclein protein aberrantly accumulates in the brains of PD and MSA patients and is thought to be one of the key drivers of pathogenesis. It is hypothesized that reduction of SNCA mRNA and, subsequently, reduced synthesis of alpha-synuclein protein will ameliorate the toxic effects of gain-of-function mutations as well as the primary pathology in PD and MSA patients without SNCA mutations.
About Parkinson’s Disease
Parkinson’s disease (PD) is a progressive neurodegenerative disease characterized by loss of neurons in the motor system. Patient’s 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 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 PD, including dominantly inherited mutations in the SNCA gene.
About Multiple System Atrophy
Multiple System Atrophy (MSA) is a rare, fatal, rapidly progressing neurodegenerative disease. Patients with MSA typically experience progressive motor dysfunction, with death often occurring less than 10 years after symptom onset. Symptoms in MSA can be similar to Parkinson’s disease, but can also include ataxia, autonomic changes and vision disturbances. Aberrant accumulation of alpha-synuclein in MSA is unique in that it is prominent not only in neurons, but also in glia cells, the supporting cells in the brain.
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