Drug intelligence / Profile preview

CpG-STAT3 siRNA

Development stage
Phase 1
Lead developer
City of Hope
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratumoral
01

Overview

CpG-STAT3 siRNA is a conjugate drug that physically links a small interfering RNA (siRNA) targeting signal transducer and activator of transcription 3 (STAT3) with a CpG oligodeoxynucleotide, a Toll-like receptor 9 (TLR9) ligand. This dual-function molecule is designed to specifically deliver siRNA into TLR9-positive immune cells and tumor cells, thereby silencing STAT3 to inhibit oncogenic and immunosuppressive signaling, while simultaneously activating innate immunity through TLR9 agonism. The drug has demonstrated preclinical and early clinical efficacy in enhancing antitumor immune responses, especially in combination with immune checkpoint inhibitors or local radiation, across models of melanoma, B-cell lymphoma, and castration-resistant prostate cancer. It is being advanced for intratumoral immunotherapy in relapsed/refractory B-cell non-Hodgkin lymphoma and under investigation for other solid and hematologic malignancies[1][2][3][4][5][6][7][8].

Other names
CpG-STAT3siRNACpG-STAT-3siRNACpG-STAT 3siRNA
02

Targets

TLR9 (Toll-like receptor 9)STAT3 (Signal Transducer and Activator of Transcription 3)

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