Drug intelligence / Profile preview

AM-21 + QTsome

Development stage
Preclinical
Lead developer
Haichang Biotech
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

AM-21 + QTsome (also known as WGI-2101 or MyMiR) is an experimental, preclinical antisense oligonucleotide therapeutic delivered via a lipid nanoparticle (LNP) platform. The active ingredient, AM-21 (antimiR-21), is a synthetic oligonucleotide designed to target and inhibit microRNA-21 (miR-21), an oncogenic microRNA (oncomiR) frequently upregulated in various human cancers, including non-small cell lung cancer (NSCLC) and colorectal cancer. QTsome is a pH-responsive LNP delivery vehicle combining quaternary amine cationic lipids and tertiary amine ionizable lipids to optimize drug loading and endosomal release. In preclinical models, AM-21 + QTsome has demonstrated significant tumor growth inhibition, upregulation of downstream miR-21 targets such as PTEN and DDAH1, and synergistic effects when combined with chemotherapeutic agents like paclitaxel or targeted therapies like erlotinib. An optimized formulation, QTPlus-AM21, has also been evaluated in combination with the immune checkpoint inhibitor atezolizumab.

Brand names
MyMiR
Other names
AM-21/QTsomeQTPlus-AM21QTPlus-AM-21QTPlus-AM 21QTsome/AM-21anti-miR-21 + QTsomeantimiR-21 + QTsome
02

Targets

miR-21 (MicroRNA-21)

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