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rAAV-shRNA targeting MRGN
- Development stage
- Preclinical
- Lead developer
- University of Florida
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intratumoral
01
Overview
rAAV-shRNA targeting MRGN is an experimental gene therapy developed by the University of Florida and Lacerta Therapeutics for the treatment of glioblastoma (GBM). The therapy utilizes a novel recombinant adeno-associated virus (rAAV) capsid, identified through AI-directed screening for high tropism to GBM cells, to deliver short hairpin RNA (shRNA) cassettes. These shRNAs are designed to target and deconstruct the Master Regulatory Gene Network (MRGN), a common regulatory state found in GBM stem-like cells (GSCs) that drives tumor heterogeneity and growth. By silencing key master regulators within this network, the therapy aims to attenuate tumor growth and improve survival in a heterogeneity-agnostic manner.
- Other names
- AAV-shRNA targeting MRGN
02
Targets
AAVR (AAV receptor)Endogenous RNA interference and microRNA machineryMRGN (Master Regulatory Gene Network)
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