ION464, also known as BIIB101, is an investigational antisense medicine designed to inhibit the production of the alpha-synuclein protein as a potential therapy for a group of neurodegenerative disorders that include Parkinson’s disease (PD) and multiple system atrophy (MSA).

About Multiple System Atrophy (MSA) and Parkinson’s Disease (PD)

Altered and insoluble forms of a protein called alpha-synuclein accumulates in the brains of PD and MSA patients and is thought to be a key driver of neurodegeneration in these diseases. It is believed that decreasing the production of the alpha-synuclein protein will reduce the accumulation and toxic effects of misfolded alpha-synuclein.

Safety and efficacy have not been evaluated by any regulatory authorities for the indication described.

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