ION582 is an investigational antisense medicine designed to inhibit the expression of the UBE3A transcript (UBE3A-ATS) for the potential treatment of Angelman syndrome (AS).
About Angelman Syndrome
AS is a rare, genetic neurological disease caused by the loss of function of the maternally inherited UBE3A gene. AS typically presents in infancy and is characterized by intellectual disability, balance issues, motor impairment, and debilitating seizures. Some patients are unable to walk or speak. Some symptoms can be managed with existing drugs; however, there are no approved disease modifying therapies.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.