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AVI-6003

Development stage: Phase 1
Lead developer: Sarepta Therapeutics
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

Overview

**AVI-6003** is an investigational, fixed-ratio antiviral combination developed by AVI BioPharma and subsequently Sarepta Therapeutics as a post-exposure countermeasure for Marburg virus disease. It comprises the PMOplus phosphorodiamidate morpholino antisense oligonucleotides AVI-7287 and AVI-7288, administered as sequential intravenous infusions at a 1:1 ratio. The components bind Marburg virus messenger RNAs encoding VP24 and nucleoprotein, respectively, to inhibit viral protein translation. AVI-6003 completed Phase 1 single-ascending-dose evaluation in healthy volunteers; subsequent Marburg development prioritized the single-agent component AVI-7288. ([ncbi.nlm.nih.gov](https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4249403/))

Targets

Marburg virus nucleoprotein messenger RNAMarburg virus VP24 protein messenger RNA

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