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 Parkinson’s disease (PD), multiple system atrophy (MSA) and related synucleinopathies.
About Multiple System Atrophy and Parkinson’s Disease
Alpha-synuclein protein abnormally accumulates in the brains of PD and MSA patients and is thought to be one of the key drivers of these diseases. It is believed that decreasing the production of the alpha-synuclein protein will reduce the toxic effects of gain-of-function mutations.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.