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RUNX1::RUNX1T1 siRNA LNP

Development stage: Preclinical
Lead developer: Princess Máxima Center for Pediatric Oncology
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, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration: Intravenous

Overview

RUNX1::RUNX1T1 siRNA LNP is a research-stage RNA interference (RNAi) therapeutic designed to treat acute myeloid leukemia (AML) harboring the t(8;21) chromosomal translocation. The therapeutic consists of chemically modified small interfering RNAs (siRNAs) that specifically target the fusion breakpoint of the RUNX1::RUNX1T1 mRNA, encapsulated within lipid nanoparticles (LNPs). To ensure selective delivery to leukemic hematopoietic cells, the LNPs are functionalized with a Leu-Asp-Val (LDV) tripeptide ligand that targets the Very Late Antigen-4 (VLA-4) integrin receptor (α4β1) commonly expressed on AML blasts. By silencing the oncogenic fusion transcript, the drug eliminates leukemic stem cell self-renewal and promotes myeloid differentiation. This compound is currently in the preclinical stage, having been developed and evaluated in patient-derived xenograft (PDX) models by academic researchers at the Princess Máxima Center for Pediatric Oncology and collaborating institutions.

Other names: LDV-functionalized RUNX1::RUNX1T1 siRNA LNPRUNX1::RUNX1T1 siRNA lipid nanoparticle

Targets

RUNX1-RUNX1T1 (Runt-related transcription factor 1; RUNX1 partner transcriptional co-repressor 1 fusion mRNA)α4β1 (Integrin α4β1)

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