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AVI-6002 + AVI-6003

Development stage: Unknown
Lead developer: Sarepta Therapeutics
Modality: 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
Administration: Intravenous

Overview

**AVI-6002 + AVI-6003** is an experimental combination therapy developed by Sarepta Therapeutics (formerly AVI BioPharma) consisting of two phosphorodiamidate morpholino oligomers (PMOplus): AVI-6002 (AVI-7537 + AVI-7539) targeting Ebola virus VP24 and VP35 viral proteins, and AVI-6003 (AVI-7287 + AVI-7288) targeting Marburg virus VP24 and NP viral proteins. These antisense oligonucleotides inhibit viral replication by sterically blocking translation of target viral mRNA. It underwent Phase 1 single-ascending-dose safety and pharmacokinetic studies in healthy volunteers, showing good tolerability up to 4.5 mg/kg per component via IV infusion, with dose-proportional PK and plasma half-life of 2-5 hours; no maximum tolerated dose was reached. Supported by DoD funding, it demonstrated efficacy in nonhuman primate models of Ebola and Marburg hemorrhagic fevers, though development shifted to single agents AVI-7537 and AVI-7288.[1][2][3]

Targets

VP35 (Ebola virus VP35)NP (Taï Forest ebolavirus nucleoprotein)

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