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AZD8701 (IONIS-1063734) is an antisense oligonucleotide targeting FOXP3 in regulatory T cells (Tregs), with a human IC50 of 65.2 nM. AZD8701 binds to intronic sites of all FOXP3 pre-mRNA isoforms and mediates dose-dependent FOXP3 knockdown via free uptake. AZD8701 can be used in cancer-related research.

For research use only. We do not sell to patients.

DNA, d(P-thio)(GATTTTGACATTCTGC) (The three nucleotides at both the 5' and 3' ends are composed of 2’?4’ cEt-modified ribonucleotides)

AZD8701 Chemical Structure

CAS No. : 2603457-08-3

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Description

AZD8701 (IONIS-1063734) is an antisense oligonucleotide targeting FOXP3 in regulatory T cells (Tregs), with a human IC50 of 65.2 nM. AZD8701 binds to intronic sites of all FOXP3 pre-mRNA isoforms and mediates dose-dependent FOXP3 knockdown via free uptake. AZD8701 can be used in cancer-related research[1][2].

In Vitro

AZD8701 (IONIS-1063734) (65 nM) potently knocks down FOXP3 in primary human Tregs (IC50 65 nM) via free uptake, reduces expression of key FOXP3 target genes by 25-50%, and impairs Treg suppressive function in vitro[1].
AZD8701 (0.001-10 μM; 9 days) potently reduces FOXP3 protein expression in primary human Tregs (EC50 = 44 nM, IC50 = 65.2 nM) and downregulates FOXP3-dependent immunosuppressive biomarkers CTLA4, ICOS, GITR, and CCR8 at 1 μM[2].
AZD8701 (1 μM; 9 days) strongly reduces FOXP3 protein expression in human thymic natural Tregs and effector Tregs subsets from healthy donor PBMCs[2].
AZD8701 reduces the suppressive function of human inducible Tregs in an in vitro effector T cell proliferation assay[2].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

In Vivo

AZD8701 induces dose-dependent FOXP3 knockdown in humanized mice, with >50% knockdown achievable at clinically feasible doses[1].
AZD8701 (6.25-50 mg/kg; s.c.; daily; 4 days) drives dose-dependent knockdown of FOXP3 mRNA and protein in human Tregs in humanized NSG mice, with significant target suppression achieved at clinically relevant doses[2].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: NOD.Cg-Prkdcscid Il2rgtm1Wjl/SzJ (NSG) (female, engrafted with 1×107 human peripheral blood mononuclear cells)[2]
Dosage: 6.25 mg/kg; 12.5 mg/kg; 25 mg/kg; 50 mg/kg
Administration: s.c.; daily; 4 days
Result: Promoted dose-dependent knockdown of FOXP3 mRNA in splenic human Tregs.
Promoted significant downregulation of FOXP3 protein levels in splenic, blood, and bone marrow human Tregs at 25 mg/kg, reducing the percentage of FOXP3+ CD4+ T cells in spleen.
Molecular Weight

5355.77

CAS No.
Appearance

Solid

Color

White to off-white

SMILES

[AZD8701]

Shipping

Room temperature in continental US; may vary elsewhere.

Storage

-20°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

Purity & Documentation
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  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

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AZD8701
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HY-159692
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