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LNP-si-LINC01257
- Development stage
- Preclinical
- Lead developer
- Children's Cancer Institute
- 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
01
Overview
LNP-si-LINC01257 is an experimental therapeutic consisting of small interfering RNA (siRNA) encapsulated within lipid nanoparticles (LNPs), specifically designed to target and silence the long non-coding RNA (lncRNA) LINC01257. Identified as a novel oncogenic driver, LINC01257 is significantly overexpressed in pediatric acute myeloid leukemia (AML) patients, particularly those harboring the t(8;21) translocation. The LNP delivery system is formulated using a lipid composition (D-Lin-MC3-DMA:DSPC:cholesterol:PEG-DMG) that reproduces the architecture of the FDA-approved siRNA drug Onpattro. Preclinical studies have demonstrated that LNP-si-LINC01257 effectively reduces cell proliferation and viability in AML cell lines while sparing healthy peripheral blood mononuclear cells, which do not express the target lncRNA. This approach represents a targeted, potentially non-toxic strategy for managing specific subtypes of pediatric leukemia.
- Other names
- siRNA-loaded lipid nanoparticles targeting LINC01257
02
Targets
LINC01257 (Long intergenic non-protein coding RNA 1257)
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