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pbi-shRNA STMN1 LP
- Development stage
- Phase 1
- Lead developer
- Gradalis
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intratumoral
01
Overview
pbi-shRNA STMN1 LP is an investigational RNA interference (RNAi) therapeutic developed by Gradalis, Inc. It consists of a plasmid encoding a bifunctional short hairpin RNA (bi-shRNA) targeting Stathmin 1 (STMN1), encapsulated in a bilamellar invaginated vesicle (BIV) lipoplex for intratumoral delivery. STMN1 is a microtubule-destabilizing protein often overexpressed in various cancers, contributing to tumor progression and resistance to taxane-based chemotherapy. By utilizing a bifunctional shRNA platform—which combines cleavage-dependent (siRNA-like) and cleavage-independent (miRNA-like) pathways—the drug achieves potent and durable knockdown of STMN1. It has been evaluated in a Phase I clinical trial for patients with advanced or metastatic solid tumors.
- Other names
- pbi-shRNA™STMN1 LPpbi-shRNA STMN1 BIV lipoplex
02
Targets
STMN1 (Stathmin-1)
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