ION717

ION717 is an investigational antisense oligonucleotide drug developed by Ionis Pharmaceuticals for the treatment of prion diseases. It is designed to reduce the expression of prion protein (PrP) in the central nervous system by targeting and degrading its messenger RNA (mRNA), thereby lowering PrP production. The drug is administered via intrathecal injection, allowing it to reach brain tissue through cerebrospinal fluid. Preclinical studies have shown that reducing PrP levels can delay disease onset, increase survival duration, and reverse neuronal damage in animal models. As of 2024, ION717 is being evaluated in a global Phase 1/2 clinical trial (NCT06153966) for symptomatic prion disease.

Other names

prion protein-targeting antisense oligonucleotidesPrP-targeting antisense oligonucleotidesPrP-lowering antisense oligonucleotide