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gemcitabine-modified microRNA-15a
- Development stage
- Preclinical
- Lead developer
- Curamir Therapeutics
- Modality
- miRNA Mimics → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → 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
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
BMI1 (Polycomb complex protein BMI-1)CHEK1 (Checkpoint kinase 1)WEE1 (WEE1 G2 checkpoint kinase)TEAD (TEAD family)YAP1 (YES-associated protein–transcriptional enhancer factor domain complex)
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