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pEzH2 + CSNPs + CD56

Development stage: Preclinical
Modality: Cell Therapies, Gene Therapies, Nanoparticles → Drug Delivery Systems, RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intravenous

Overview

pEzH2 + CSNPs + CD56 is a targeted gene delivery and immunotherapy nanosystem designed for the treatment of acute myeloid leukemia (AML). The formulation consists of a plasmid encoding short hairpin RNA targeting Enhancer of Zeste Homolog 2 (EZH2)—a key epigenetic regulator implicated in cancer—loaded into chitosan nanoparticles (CSNPs) and further functionalized with a CD56-targeting moiety. CD56 is a surface marker highly expressed on natural killer (NK) cells, and targeting this marker enables selective delivery to (or engineering of) NK cells for anti-leukemic activity. The therapeutic modality involves in vivo genetic engineering of NK cells to enhance immune-mediated destruction of AML cells by silencing EZH2 expression, thereby compromising leukemic cell survival and proliferation. This system exploits nanoparticle delivery for improved gene transfer, enhanced targeting, and minimal off-target effects[6].

Other names: pEzH2@CsNPs@CD56

Targets

NCAM1 (Neural cell adhesion molecule 1)EZH2 (Histone-lysine N-methyltransferase EZH2)

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