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CEP55-targeted antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- QIMR Berghofer Medical Research Institute
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
CEP55-targeted antisense oligonucleotide is an experimental RNA-based therapy designed to inhibit the expression of Centrosomal protein 55 (CEP55), an oncogenic driver overexpressed in various aggressive and therapy-resistant cancers. The drug is formulated using tumor-antigen targeted lipid nanoparticles (ASO-LNP) to facilitate delivery. By silencing CEP55, the therapy disrupts the PI3K/AKT and ERK signaling pathways, remodels the tumor microenvironment (including extracellular matrix architecture and integrin trafficking), and activates anti-proliferative stress responses. Preclinical studies in mouse models of lung adenocarcinoma and PTEN-deficient tumors have demonstrated that CEP55 inhibition suppresses tumor growth and extends survival.
- Other names
- CEP55-targeting ASO-LNPCEP-55-targeting ASO-LNPCEP 55-targeting ASO-LNPCEP55 ASOCEP-55 ASOCEP 55 ASO
02
Targets
CEP55 (Centrosomal protein 55)
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