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CpG-aptaDiR

Development stage: Preclinical
Lead developer: Aptadir Therapeutics
Modality: RNA Aptamers → 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, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intravenous

Overview

CpG-aptaDiR is a bi-functional synthetic oligonucleotide conjugate designed for the targeted epigenetic immunotherapy of acute myeloid leukemia (AML). It consists of a TLR9-activating CpG oligodeoxynucleotide linked to a DNA methyltransferase 1 (DNMT1)-neutralizing RNA aptamer (aptaDiR). This platform utilizes the CpG moiety to achieve myeloid-selective delivery via TLR9-mediated internalization, while the aptamer component inhibits DNMT1 activity to reverse epigenetic suppression of leukemic differentiation and antigen presentation. By combining DNMT1 inhibition with innate immunostimulation, CpG-aptaDiR aims to eliminate leukemia progenitor/stem cells (LSCs) and enhance antitumor immunity, potentially overcoming resistance to standard hypomethylating agents.

Other names: CpG-DNMT1 aptamerCpG-DNMT-1 aptamerCpG-DNMT 1 aptamerCpG-DNMT1 aptCpG-DNMT-1 aptCpG-DNMT 1 aptTLR9 agonist–DNMT1 aptamer conjugate

Targets

DNMT1 (DNA (cytosine-5)-methyltransferase 1)TLR9 (Toll-like receptor 9)

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