Drug intelligence / Profile preview

gemcitabine-modified microRNA-15a

Development stage
Preclinical
Lead developer
Curamir Therapeutics
Modality
miRNA Mimics → 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
01

Overview

Gemcitabine-modified microRNA-15a (Gem-miR-15a) is a novel nucleic acid-based therapeutic developed by researchers at Stony Brook University and Curamir Therapeutics. It is an engineered microRNA (miRNA) mimic where the endogenous cytidine bases in the guide strand of miR-15a are replaced with the nucleoside analog gemcitabine. This chemical modification significantly enhances the stability and potency of the miRNA. Gem-miR-15a functions as a multi-targeted agent, downregulating several key oncogenic proteins including WEE1, CHK1, BMI1, and YAP1. In preclinical studies for pancreatic ductal adenocarcinoma (PDAC) and colorectal cancer (CRC), the drug has demonstrated the ability to induce S-phase cell cycle arrest and apoptosis, showing high cytotoxicity in both parental and chemoresistant cell lines and effectively inhibiting tumor growth in vivo without significant toxicity.

Other names
Gem-modified miR-15agemcitabine-modified miR-15a
02

Targets

CHEK1 (Checkpoint kinase 1)BMI1 (Polycomb complex protein BMI-1)WEE1 (WEE1 G2 checkpoint kinase)TEAD (TEAD family)

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