Drug intelligence / Profile preview

c-myc antisense oligonucleotide

Development stage
Unknown
Lead developer
Inex
Modality
Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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
Administration
Intravenous, Intracoronary, Local (tumor), Subcutaneous (via Microsphere Delivery In Animal Models)
01

Overview

A **c-myc antisense oligonucleotide** is a synthetic strand of nucleic acid designed to specifically bind to the mRNA of the c-myc proto-oncogene, inhibiting its translation and thereby reducing c-myc protein expression. c-myc is a well-established oncogene whose overexpression drives tumor growth, proliferation, and survival in many cancers. Mechanistically, these antisense oligonucleotides (ASOs) work by hybridizing to target c-myc mRNA, leading to its degradation or blocking its translation, thus downregulating oncogenic Myc protein levels. Early clinical candidates included phosphorothioate oligodeoxynucleotides (e.g., INX-3280) and phosphorodiamidate morpholino oligomers (e.g., AVI-4126/Resten-RG), as well as more recent locked nucleic acid-based gapmers. Development has focused on cancer (lymphoma, solid tumors, leukemia, melanoma, hepatocellular carcinoma) and vascular restenosis, though no candidate has reached market approval. Delivery approaches have included intravenous, local/intracoronary, and microencapsulated sustained-release formulations[1][2][3][4][5].

Brand names
Resten-RGResten-NG
Other names
MYC antisense oligonucleotidec-myc ASOc-myc ODNc-myc antisense oligodeoxynucleotide
02

Targets

MYC (MYC proto-oncogene protein)

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