Drug intelligence / Profile preview

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, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → 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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