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siMETTL3
- Development stage
- Preclinical
- Lead developer
- STORM Therapeutics
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
siMETTL3 is a nanoparticle-based small interfering RNA (siRNA) therapeutic designed to target and silence the expression of methyltransferase-like 3 (METTL3). METTL3 is the primary catalytic subunit of the RNA methyltransferase complex responsible for N6-methyladenosine (m6A) modification, an epigenetic mark that regulates mRNA stability, translation, and splicing. The therapeutic utilizes a specific siRNA sequence (sense 5'-CCAAGGAAGAGUGCAUGAATT-3', antisense 5'-UUCAUGCACUCUUCCUUGGTT-3') modified with 2'-O-methylation for enhanced stability and is delivered via PEG-PLGA-based vesicle-like nanoparticles (VNP). In preclinical models of non-alcoholic fatty liver disease (NAFLD)-related hepatocellular carcinoma, siMETTL3 has demonstrated the ability to induce antitumor immune responses and synergize with anti-PD-1 immunotherapy by increasing tumor-infiltrating CD8+ T cells. It has also been investigated for its role in modulating aberrant androgen receptor signaling in prostate cancer.
- Other names
- METTL3 siRNAMETTL-3 siRNAMETTL 3 siRNAsiRNA-METTL3siRNA-METTL-3siRNA-METTL 3
02
Targets
METTL3 (Methyltransferase-like protein 3)
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