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hIRF4-ASO#3
- Development stage
- Preclinical
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intraperitoneal
01
Overview
hIRF4-ASO#3 is a human-specific antisense oligonucleotide (ASO) designed to selectively target and inhibit the expression of Interferon Regulatory Factor 4 (IRF4). IRF4 is a transcription factor critical for the survival and proliferation of various hematologic malignancies, including diffuse large B-cell lymphoma (DLBCL) and multiple myeloma. By binding to IRF4 mRNA, hIRF4-ASO#3 triggers its degradation, thereby reducing IRF4 protein levels and suppressing downstream oncogenic signaling. Preclinical research has demonstrated that hIRF4-ASO#3 can impair lymphomagenesis and resensitize BCL2-overexpressing DLBCL cells to chemotherapy agents such as doxorubicin and venetoclax (ABT-199). While hIRF4-ASO#3 is primarily utilized as a research tool compound to validate IRF4 as a therapeutic target, its development is closely linked to clinical-stage candidates like ION251.
- Other names
- human IRF4-specific antisense oligonucleotide #3
02
Targets
IRF4 (Interferon regulatory factor 4)
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