Chemistry: Generation 2+
ION716 is an investigational antisense medicine targeting prion protein (PrP) messenger ribonucleic acid (mRNA). ION716 is designed to inhibit the production of cellular PrP protein (PrPC) and is being developed as a potential therapy for Prion diseases. ASO-mediated reduction of PrPC has the potential to ameliorate, prevent, or even reverse Prion diseases.
About Prion Diseases
Prion disease is a fatal, incurable neurodegenerative disease that typically presents as a rapidly progressive dementia. Regardless of etiology – sporadic, genetic, or acquired, and regardless of clinical name – Creutzfeldt-Jakob disease, fatal familial insomina, or Gerstmann-Straussler-Scheinker syndrome, all prion diseases are caused by conformational change of PrP from its native fold to a self-propagating misfolded form (PrPSc), which is an abnormal, pathogenic agent that causes brain damage.