Drug intelligence / Profile preview

miR-124-PD-1

Development stage
Preclinical
Modality
Cell Therapies, Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intratumoral, Intracranial, Intravenous, Intrathecal
01

Overview

miR-124-PD-1 is an experimental dual-gene therapy platform that combines microRNA-124 (miR-124), a tumor-suppressive microRNA, with programmed cell death protein 1 (PD-1) modulation. The approach typically involves the co-delivery of both miR-124 and PD-1 genes using vectors such as umbilical cord mesenchymal stem cells and exosomes. miR-124 is known to inhibit tumor proliferation, promote apoptosis, suppress malignant transformation, and enhance sensitivity to anti-tumor drugs by targeting multiple oncogenic pathways. PD-1 modulation aims to enhance anti-tumor immune responses by blocking immune checkpoint pathways that tumors exploit for immune evasion. This combination strategy is being investigated primarily for glioblastoma therapy but may have broader applications in oncology[7][6][2].

02

Targets

ANXA5CDK6 (Cyclin-dependent kinase 6)PDCD1 (Programmed cell death protein 1 receptor)

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