Drug intelligence / Profile preview

pSil-miR200c

Development stage
Phase 1
Modality
miRNA Mimics → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, miRNA Inhibitors → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies
Administration
Intratumoral (experimental), Intravenous (experimental), Systemic (via Nanoparticle Delivery)
01

Overview

pSil-miR200c is a gene therapy construct designed to deliver and overexpress microRNA-200c (miR-200c), a member of the miR-200 family of microRNAs. miR-200c regulates gene expression post-transcriptionally by binding to target mRNAs, leading to their degradation or translational repression[1]. The miR-200 family plays a critical role in inhibiting epithelial-mesenchymal transition (EMT), which is an initiating step in cancer metastasis, by targeting transcriptional repressors such as ZEB1 and ZEB2 that downregulate E-cadherin[2][5][6]. Overexpression of miR-200c has been shown to suppress tumor progression, invasion, migration, stem-like phenotype, and proliferation in various cancers including breast cancer and ovarian cancer[5][6]. Additionally, miR-200c modulates apoptosis pathways—generally promoting apoptosis—and influences immune responses by regulating PD-L1 expression and myeloid-derived suppressor cell activity[4]. Delivery systems for therapeutic use include nanoparticle-based carriers for targeted delivery into tumor cells[5].

02

Targets

CD274 (Programmed cell death protein 1 ligand 1)ZEB1 (Zinc finger E-box-binding homeobox 1)ZEB2 (Zinc finger E-box-binding homeobox 2)

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