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avi-4126

Development stage: Phase 2
Lead developer: Sarepta Therapeutics
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
Administration: Intravenous, Intracoronary

Overview

AVI-4126 is a third-generation antisense phosphorodiamidate morpholino oligomer (PMO) that targets the c-myc mRNA, blocking its translation and thereby inhibiting the production of the c-MYC protein. This mechanism can result in antineoplastic effects by inducing death in tumor cells overexpressing c-MYC and has also been investigated for its ability to inhibit cell proliferation involved in cardiovascular restenosis. Originally developed by AVI BioPharma (now Sarepta Therapeutics), AVI-4126 was studied for cancer, coronary artery restenosis, and other indications but development has been discontinued for these diseases[1][2][4][5][7].

Brand names: RESTEN-MP
Other names: phosphorodiamidate morpholino oligomer AVI-4126

Targets

MYC (MYC proto-oncogene protein)

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