Drug intelligence / Profile preview

LOR-1284

Development stage
Preclinical
Lead developer
Aptose Biosciences
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Sublingual
01

Overview

**LOR-1284** is a small interfering RNA (siRNA) therapeutic candidate developed by Lorus Therapeutics Inc. that specifically targets the R2 subunit of ribonucleotide reductase (RRM2), a key enzyme in de novo DNA synthesis essential for cancer cell proliferation. By silencing RRM2 expression, LOR-1284 inhibits tumor growth and has demonstrated strong preclinical antitumor activity in xenograft models of human cancers, including renal cell carcinoma, melanoma, and acute myeloid leukemia (AML). It has been formulated in novel lipid nanoparticles, such as transferrin-lipid nanoparticles and proteinase K-containing nanoparticles, to enhance delivery and overcome drug resistance, with applications explored in combination with commercial chemotherapeutics.[1][3][5][7][9][11]

02

Targets

RRM2

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