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Smyd3 ASO
- Development stage
- Unknown
- Lead developer
- Ionis Pharmaceuticals
- 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
- Unknown
01
Overview
Smyd3 ASO is a next-generation antisense oligonucleotide therapy designed to silence expression of the SMYD3 gene, which encodes an oncogenic lysine methyltransferase frequently overexpressed in hepatocellular carcinoma and other malignancies. This drug utilizes ASO technology to specifically target and degrade Smyd3 mRNA, reducing tumor growth, inhibiting oncofetal and cancer stem cell-specific gene activation, and blocking cellular de-differentiation associated with liver cancer. Smyd3 ASO has demonstrated efficacy in mouse models and human hepatic cancer cell lines, notably decreasing tumor burden and preventing activation of cancer-promoting gene networks.
02
Targets
SMYD3 (SET and MYND domain-containing protein 3)
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