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Drug intelligence / Profile preview
pEzH2 + CSNPs + CD56
- Development stage
- Preclinical
- Modality
- Cell Therapies, Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
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
02
Targets
NCAM1 (Neural cell adhesion molecule 1)EZH2 (Histone-lysine N-methyltransferase EZH2)
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