Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
AVI-4020
- Development stage
- Phase 1
- 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-4020 is an antisense phosphorodiamidate morpholino oligomer (PMO) drug candidate developed for the treatment of West Nile virus (WNV) infection, specifically targeting patients with acute neuroinvasive disease. It was designed using NeuGene technology to bind to the translation start site of the single open reading frame of WNV, thereby inhibiting viral replication. The compound has demonstrated the ability to cross the blood-brain barrier and reduce viral titers in preclinical studies. Clinical trials included a Phase 1 study evaluating safety, tolerability, pharmacokinetics, and potential effectiveness in hospitalized patients with presumptive acute neuroinvasive WNV disease. Although AVI-4020 showed promise in early studies and was safe to administer, its development was ultimately discontinued.
02
Targets
WNV (West Nile virus)
Beyond the preview
Go deeper on AVI-4020.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on AVI-4020.