Drug intelligence / Profile preview

si-HPS4

Development stage
Preclinical
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
Intravenous, Intratumoral
01

Overview

si-HPS4 is a small interfering RNA (siRNA) designed to specifically silence the expression of the HPS4 (Hermansky-Pudlak Syndrome 4) gene. HPS4 is a critical component of the Biogenesis of Lysosome-related Organelles Complex 3 (BLOC-3), which functions as a guanine nucleotide exchange factor (GEF) for the Rab GTPases Rab27a and Rab32/38, thereby regulating the trafficking of proteins to lysosomes and lysosome-related organelles. In the context of oncology, particularly liver hepatocellular carcinoma (LIHC), HPS4 has been identified as a potential oncogene and a novel therapeutic target. Overexpression of HPS4 is associated with poor prognosis and altered immune infiltration in liver cancer. By utilizing the RNA interference (RNAi) pathway, si-HPS4 mediates the degradation of HPS4 mRNA, leading to reduced protein levels and subsequent inhibition of cancer cell proliferation and progression. It is currently utilized in preclinical research to validate the therapeutic potential of HPS4 inhibition.

Other names
HPS4 siRNAHPS-4 siRNAHPS 4 siRNAsmall interfering RNA targeting HPS4siRNA-HPS4siRNA-HPS-4siRNA-HPS 4
02

Targets

HPS4 (Hermansky-Pudlak syndrome 4 protein)

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