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ISIS 14803
- Development stage
- Phase 2
- Lead developer
- Ionis Pharmaceuticals
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous, Subcutaneous
01
Overview
ISIS 14803 is a 20-unit antisense phosphorothioate oligodeoxynucleotide developed as an inhibitor of hepatitis C virus (HCV). It binds specifically to the HCV RNA at the translation initiation region of the internal ribosome entry site (IRES), thereby inhibiting viral protein expression and replication in cell culture and animal models. The drug was investigated primarily for chronic HCV infection, including in patients resistant to other therapies. Clinical trials demonstrated some antiviral activity, with transient reductions in plasma HCV RNA observed in a minority of patients, often accompanied by asymptomatic elevations in liver enzymes. The development program included both intravenous and subcutaneous administration routes.
02
Targets
HCV IRES (Hepatitis C virus internal ribosome entry site)
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