Drug intelligence / Profile preview

siRNA targeting GGH

Development stage
Preclinical
Lead developer
National Defense Medical Center
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous
01

Overview

siRNA targeting GGH is an experimental RNA interference (RNAi) therapeutic designed to silence the expression of gamma-glutamyl hydrolase (GGH). GGH is an enzyme involved in folylpolygamma-glutamate metabolism and has been identified as a prognostic biomarker and driver of malignancy in glioblastoma multiforme (GBM). By knocking down GGH, the siRNA disrupts proliferative stem-tumor cells, induces membrane disruption and necrotic changes, and inhibits tumor growth. Research indicates that GGH knockdown affects oxidative phosphorylation, MYC regulation, and reactive oxygen species (ROS) pathways, making it a potential target for treating resistant GBM.

Other names
GGH siRNAsiRNA-GGH
02

Targets

GGH (γ-glutamyl hydrolase)

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