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Drug intelligence / Profile preview
anti-MGMT morpholino oligonucleotides
- Development stage
- Preclinical
- Lead developer
- Oregon Health & Science University
- 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
- Administration
- Intratumoral, Intravenous
01
Overview
Anti-MGMT morpholino oligonucleotides (AMONs) are a class of antisense oligonucleotide research compounds designed to silence the DNA repair enzyme O6-methylguanine DNA methyltransferase (MGMT). MGMT is a primary mechanism of resistance to alkylating chemotherapies, such as temozolomide, in glioblastoma and other solid tumors. AMONs utilize a neutral phosphorodiamidate morpholino backbone, which provides high stability and target affinity compared to traditional ribose-based oligonucleotides. By binding to MGMT mRNA, they block translation, reducing MGMT protein levels and sensitizing tumor cells to chemotherapy. Research at Oregon Health & Science University (OHSU) has also explored using sub-lethal ionizing radiation to enhance the intracellular uptake of these oligonucleotides in tumor cells.
- Other names
- anti-MGMT morpholinosMGMT morpholino oligonucleotides
02
Targets
MGMT (O6-methylguanine-DNA methyltransferase)AGT (O6-methylguanine-DNA methyltransferase)
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