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GTI-3611 is an antisense oligonucleotide designed to target the messenger RNA (mRNA) of the R2 subunit of ribonucleotide reductase (RNR). Ribonucleotide reductase is a critical enzyme in DNA synthesis, responsible for converting ribonucleotides into deoxyribonucleotides. By inhibiting the expression of the R2 subunit (RRM2), GTI-3611 aims to deplete the intracellular pool of deoxyribonucleotides required for DNA replication and repair, thereby inhibiting tumor cell proliferation and potentially sensitizing cells to other chemotherapeutic agents. Developed by Lorus Therapeutics (now Aptose Biosciences), GTI-3611 was primarily investigated in preclinical and early clinical settings for the treatment of various solid tumors. Although it demonstrated efficacy in several cancer models, development eventually shifted toward a related lead candidate, GTI-2040 (later known as LOR-2040).
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