Drug intelligence / Profile preview

HMMR shRNA

Development stage
Preclinical
Lead developer
Kennedy Krieger Institute
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratumoral
01

Overview

HMMR shRNA is a gene-silencing therapeutic candidate designed to target the hyaluronan-mediated motility receptor (HMMR), also known as RHAMM. In the context of glioblastoma, HMMR is hyper-expressed and correlates with poor patient survival. The drug utilizes lentivirus-mediated delivery of short hairpin RNA to potently knockdown HMMR expression in glioblastoma stem cells (GSCs). This knockdown disrupts GSC self-renewal, inhibits the expression of stemness markers such as CD133, SOX2, and Olig2, and attenuates Notch signaling activity. Preclinical studies have demonstrated that HMMR knockdown blocks the in vivo tumorigenic potential of GSCs in mouse orthotopic xenograft models, suggesting its potential as a therapeutic strategy for depleting tumor-initiating cells in glioblastoma.

Other names
RHAMM shRNAhyaluronan-mediated motility receptor shRNA
02

Targets

HMMR

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