Drug intelligence / Profile preview

GTI-2040

Development stage
Phase 2
Lead developer
Aptose Biosciences
Modality
Small Molecules, 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

GTI-2040 is an investigational antisense oligonucleotide designed to inhibit the R2 subunit of ribonucleotide reductase, an enzyme essential for DNA synthesis and cell proliferation. By binding to the mRNA encoding the R2 component, GTI-2040 decreases both mRNA and protein levels of R2, thereby potentially inhibiting tumor cell proliferation. The R2 subunit is often elevated in various cancers and contributes to tumor growth and metastatic potential. Preclinical studies have shown activity against multiple tumor types, including colon, liver, lung, breast, kidney, and ovary cancers. GTI-2040 has been evaluated in clinical trials for several malignancies as monotherapy or in combination with other chemotherapeutic agents[1][3][4][5].

02

Targets

RRM2

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