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GTI-2601
- Development stage
- Discontinued
- Lead developer
- Aptose Biosciences
- 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
01
Overview
GTI-2601 is a phosphorothioate antisense oligonucleotide (PS-AS-ODN) developed by Lorus Therapeutics (now Aptose Biosciences) for the potential treatment of cancer. It specifically targets the mRNA of human thioredoxin (Trx), a protein that is frequently overexpressed in various tumors and acts as a growth stimulator and inhibitor of apoptosis. By binding to thioredoxin mRNA, GTI-2601 facilitates its degradation, thereby reducing the levels of thioredoxin protein within the cell. Preclinical studies demonstrated that GTI-2601 exhibits potent anti-proliferative effects on colony formation in vitro and significant anti-tumor activity in human tumor xenograft mouse models in vivo. Despite promising early results, development of the compound appears to have been discontinued in the preclinical or early clinical stages.
- Other names
- Trx-ASthioredoxin antisense oligonucleotide
02
Targets
TXN (Thioredoxin-1)
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