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Drug intelligence / Profile preview
CpG-siRNARPUSD4
- Development stage
- Preclinical
- 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
01
Overview
**CpG-siRNARPUSD4** is a novel RNA interference therapeutic consisting of a CpG oligodeoxynucleotide conjugated to a small interfering RNA (siRNA) targeting RPUSD4, a mitochondrial RNA pseudouridine synthase. RPUSD4 modifies mitochondrial RNA, reprogramming cancer cell metabolism to support tumor growth. The CpG moiety acts as a Toll-like receptor 9 (TLR9) agonist, stimulating innate immune activation in antigen-presenting cells, while the siRNA silences RPUSD4 expression, disrupting mitochondrial function and inducing tumor cell death. Preclinical studies in acute myeloid leukemia (AML) models demonstrate significant survival prolongation upon RPUSD4 pharmacological targeting, positioning this conjugate as a potential dual immunomodulatory and metabolic inhibitor for oncology applications.[3]
- Other names
- CpG-conjugated RPUSD4 siRNA
02
Targets
TLR9 (Toll-like receptor 9)RPUSD4 (RNA pseudouridine synthase D4)
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