Drug intelligence / Profile preview

TargomiRs

Development stage
Phase 1
Lead developer
Engeneic
Modality
Gene Therapies, Small Interfering RNA (siRNA) → Small RNA Therapeutics → 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, Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

TargomiRs are a novel therapeutic platform consisting of nonliving bacterial minicells (nanoparticles) known as EnGeneIC Dream Vectors, which are loaded with a synthetic microRNA (miR-16-based mimic) and targeted to tumors via an epidermal growth factor receptor (EGFR)-specific antibody. The primary mechanism is the restoration of tumor-suppressive miR-15/miR-16 family function in cancer cells where these microRNAs are downregulated, leading to inhibition of oncogenic targets such as BCL2, CDK1, and JUN. The minicell delivery system protects the RNA payload from degradation and enables targeted delivery to EGFR-expressing tumor cells. TargomiRs have been primarily developed for malignant pleural mesothelioma but may have broader applications in oncology.

Other names
nanocell-encapsulated miR-16-based microRNA mimicEnGeneIC Dream Vectors loaded with miR-16 mimic
02

Targets

BCL-2 (BCL-2 family)Cyclin-dependent kinase 1 (CDK1) mRNAJUN (Transcription factor Jun)CDK1 (Cyclin-dependent kinase 1)FOS (Activator Protein 1)

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