ION717 is an investigational antisense medicine designed to inhibit the production of prion protein (PrP) for the potential treatment of prion disease.
About Prion Disease
Prion disease is a rare, fatal neurodegenerative disease caused by misfolding of PrP which accumulates in the brain. People with prion disease often experience progressive memory impairment, personality changes, difficulties with movement and loss of independence. There are currently no effective disease-modifying treatments for prion disease. In most cases, a person succumbs to prion disease within a year following symptom onset.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.