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AVI-5126
- Development stage
- Preclinical
- 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, Small Molecules, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intracoronary, Ophthalmic
01
Overview
AVI-5126 is a phosphorodiamidate morpholino oligomer (PMO) antisense drug developed by AVI BioPharma as part of its NeuGene platform. Its primary mechanism of action is the inhibition of DNA expression, specifically targeting and suppressing the proto-oncogene c-myc, which plays a key role in transplant rejection and vascular proliferative disorders. The drug was investigated for use in preventing coronary artery restenosis—re-narrowing of blood vessels after interventions such as angioplasty or coronary artery bypass graft (CABG) surgery—and was also studied preclinically for delaying corneal allograft rejection by inhibiting c-myc expression. Clinical development reached Phase II trials for coronary artery restenosis but was discontinued[2][4][7]. Preclinical studies demonstrated that AVI-5126 could delay graft rejection in corneal transplantation models without significant toxicity[3][5][6].
- Brand names
- Restin-CP
- Other names
- NeuGene antisense compound
02
Targets
MYC (MYC proto-oncogene protein)
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