ION541, also known as BIIB105, is an investigational antisense medicine designed to reduce the production of the ataxin-2 (ATXN2) protein for the potential treatment of amyotrophic lateral sclerosis (ALS). The reduction of ATXN2 has been shown to decrease aggregation of TDP-43, a toxic RNA binding protein found in most patients with ALS, including the approximately 90 percent of the ALS population with no known family history of ALS.
About Amyotrophic Lateral Sclerosis
ALS is a rare, fatal neurodegenerative disorder characterized by progressive degeneration of motor neurons resulting in a declining quality of life due to muscle weakness, loss of movement and difficulty in breathing and swallowing. Currently, treatment options for patients with ALS are extremely limited with no drugs that significantly slow disease progression.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.