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AVI-4557
- Development stage
- Unknown
- Lead developer
- Sarepta Therapeutics
- Modality
- Small Molecules, 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
01
Overview
AVI-4557 is an antisense phosphorodiamidate morpholino oligomer (PMO) drug developed by Sarepta Therapeutics (formerly AVI BioPharma) to selectively inhibit the expression of cytochrome P450 3A4 (CYP3A4). CYP3A4 is a key liver enzyme responsible for the metabolism of approximately half of all marketed drugs. By silencing CYP3A4, AVI-4557 reduces the metabolic breakdown of co-administered drugs, resulting in increased blood concentrations and prolonged drug action. This mechanism was explored to potentially limit toxicity or enhance efficacy for patients receiving highly toxic therapeutic agents, such as those used in anxiety or cancer treatment. The compound underwent several Phase I clinical trials demonstrating effective downregulation of CYP3A4 with good safety and tolerability profiles. Development appears discontinued.
- Other names
- NEUGENE antisense drug
02
Targets
CYP3A4 (Cytochrome P450 3A4)Cytochrome P450 family 3 subfamily A member 4 mRNA
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