Drug intelligence / Profile preview

TargomiRs

Development stage
Phase 1
Lead developer
Engeneic
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Addition/Replacement → Gene Therapies, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Gene Silencing → Gene Therapies, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies, Nanoparticles → Drug Delivery Systems, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
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

JUN (Transcription factor Jun)Cyclin-dependent kinase 1 (CDK1) mRNAApoptosis regulator Bcl-2 mRNA

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