Drug intelligence / Profile preview

NPM1 small interfering RNA

Development stage
Preclinical
Lead developer
Thermo Fisher Scientific
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
Intravenous, Intratumoral
01

Overview

NPM1 small interfering RNA (siRNA) is a targeted molecular therapeutic designed to silence the expression of the Nucleophosmin (NPM1) gene through the RNA interference (RNAi) pathway. NPM1 is a multifunctional nucleolar protein that plays critical roles in ribosome biogenesis, centrosome duplication, and genomic stability; however, its overexpression or mutation (particularly the NPM1c+ mutation) is a hallmark of various malignancies, including acute myeloid leukemia (AML) and several solid tumors. By introducing synthetic double-stranded RNA sequences complementary to the NPM1 mRNA, this agent facilitates the assembly of the RNA-induced silencing complex (RISC), which subsequently cleaves the target mRNA and prevents protein translation. Preclinical studies have demonstrated that silencing NPM1 can inhibit cell proliferation, induce apoptosis, and enhance the sensitivity of cancer cells to conventional chemotherapeutic agents, making it a significant area of investigation in oncology.

Other names
Nucleophosmin siRNANPM siRNANPM1 siRNANPM-1 siRNANPM 1 siRNA
02

Targets

NPM1 (Nucleophosmin)

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