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anti-CD38-targeted lipid nanoparticles delivering small interfering RNA targeting CKAP5

Development stage
Preclinical
Lead developer
Tel Aviv University
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, Antibody-Based Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

This experimental RNA therapeutic is a targeted delivery system designed for the treatment of multiple myeloma. It consists of lipid nanoparticles (LNPs) formulated with a novel ionizable lipid and coated with an anti-CD38 antibody to specifically target CD38-expressing malignant plasma cells in the bone marrow. The therapeutic payload is a small interfering RNA (siRNA) directed against cytoskeleton-associated protein 5 (CKAP5), a protein essential for microtubule stability and spindle assembly during the cell cycle. By silencing CKAP5, the drug induces cell cycle arrest and significantly reduces the viability of multiple myeloma cells. Preclinical studies have demonstrated that this targeted approach achieves high therapeutic efficacy in xenograft mouse models while reducing off-target effects, such as hepatic accumulation, which is a common limitation of non-targeted LNP delivery systems.

Other names
anti-CD38-targeted lipid nanoparticles delivering siRNA targeting CKAP5anti-CD-38-targeted lipid nanoparticles delivering siRNA targeting CKAP5anti-CD 38-targeted lipid nanoparticles delivering siRNA targeting CKAP5CD38-targeted LNPs delivering siRNA-CKAP5CD-38-targeted LNPs delivering siRNA-CKAP5CD 38-targeted LNPs delivering siRNA-CKAP5αCD38-tLNPs-siRNA-CKAP5
02

Targets

CD38 (Cluster of Differentiation 38)

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