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ION716
- Development stage
- Discontinued
- Lead developer
- Ionis Pharmaceuticals
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
ION716 is an investigational **antisense oligonucleotide** drug candidate developed by **Ionis Pharmaceuticals** for **prion disease**. It was designed to bind **PRNP mRNA** and reduce production of the prion protein in the central nervous system, with the therapeutic goal of lowering brain **PrP** levels in prion disorders. The program used an **intrathecally administered** antisense approach, but Ionis later deprioritized this candidate in favor of a different prion ASO with improved properties, and the company subsequently advanced **ION717** instead.
02
Targets
PRNP (Cellular Prion Protein)
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