Drug intelligence / Profile preview

CpG oligodeoxynucleotide + scrambled RNA

Development stage
Preclinical
Lead developer
City of Hope Comprehensive Cancer Center
Modality
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
Administration
Intravenous
01

Overview

CpG oligodeoxynucleotide + scrambled RNA (also known as CpG-scrRNA) is a synthetic bi-functional oligonucleotide conjugate designed as a negative control for CpG-aptaDiR, an epigenetic immunotherapy for acute myeloid leukemia (AML). The molecule consists of a Toll-like receptor 9 (TLR9)-activating CpG oligodeoxynucleotide covalently linked to a scrambled RNA sequence that lacks specific biological activity. While the CpG component provides innate immunostimulation via TLR9 agonism, the scrambled RNA serves as a non-functional structural analog to the DNMT1-inhibiting aptamer found in the lead therapeutic. In preclinical studies conducted by researchers at City of Hope, CpG-scrRNA is utilized to demonstrate that the anti-leukemic effects of CpG-aptaDiR—such as the elimination of leukemia stem cells and induction of differentiation—are dependent on the specific targeting of DNMT1 rather than general TLR9 activation alone.

Other names
inactive CpG-scrRNA
02

Targets

TLR9 (Toll-like receptor 9)

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