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ISIS 5320
- Development stage
- Preclinical
- Lead developer
- ImQuest Pharmaceuticals
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Topical
01
Overview
ISIS 5320 is a phosphorothioate oligonucleotide developed as an anti-HIV agent. It acts by a novel non-antisense mechanism, forming a stable tetrameric G-quartet structure that directly binds to the V3 loop of the HIV envelope glycoprotein gp120. This interaction blocks the binding of gp120 to the CD4 receptor on human cells, thereby preventing both virus-to-cell and cell-to-cell transmission of HIV. The compound has demonstrated potent inhibition of HIV infection in vitro against various laboratory and clinical isolates, including drug-resistant strains. It was investigated primarily as a topical microbicide for prevention of sexual transmission of HIV[1][2][4].
- Other names
- TTGGGGTT
02
Targets
gp120 (HIV-1 envelope glycoprotein (gp120/gp41) epitopes)
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