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AVI-6002
- Development stage
- Phase 1
- 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
01
Overview
AVI-6002 is an experimental combination drug developed for the treatment and post-exposure prophylaxis of Ebola virus infection. It consists of two phosphorodiamidate morpholino oligomers (PMOs), specifically AVI-7537 and AVI-7539, which are designed to target the viral matrix proteins VP24 and VP35 by binding to their mRNA sequences and blocking translation. This mechanism inhibits viral replication. The PMOs in AVI-6002 use a proprietary chemistry (PMOplus) that incorporates positively charged piperazine residues to enhance binding affinity for negatively charged viral RNA, potentially reducing the risk of resistance mutations. The drug is administered intravenously and has shown efficacy in non-human primate models as well as safety in phase 1 clinical trials in humans[2][3][4][5][7].
- Other names
- AVI-002AVI002AVI 002Avipero
02
Targets
VP35 (Ebola virus VP35)
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