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AVI-4065
- Development stage
- Phase 1
- Lead developer
- Sarepta Therapeutics
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → 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, Small Molecules
- Administration
- Intravenous
01
Overview
AVI-4065 is a phosphorodiamidate morpholino oligomer (PMO) antisense oligonucleotide developed as an investigational antiviral drug for the treatment of hepatitis C virus (HCV) infection. It was designed to inhibit HCV replication by irreversibly binding to the viral genome, thereby preventing viral protein synthesis and subsequent virus replication. The drug was developed using proprietary NeuGene antisense technology and underwent early-phase clinical trials that demonstrated favorable safety and tolerability profiles but did not achieve the therapeutic threshold required for efficacy at tested doses. Development of AVI-4065 for hepatitis C has been discontinued[1][2][4][6][7].
02
Targets
HCV RNA (Hepatitis C virus RNA)
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