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DVAP-Exo + mitoxantrone + Notch1 siRNA
- Development stage
- Preclinical
- Lead developer
- China Pharmaceutical University
- Modality
- Small Molecules, 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, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
DVAP-Exo + MIT + siRNA targeting Notch1 is an experimental, exosome-based co-delivery system designed for the targeted treatment of liver fibrosis. The formulation consists of exosomes surface-modified with the DVAP peptide (a D-peptide ligand that specifically targets Vascular Adhesion Protein-1, VAP-1, which is overexpressed on activated hepatic stellate cells). These engineered exosomes encapsulate mitoxantrone (MIT), a cytotoxic small molecule, and a small interfering RNA (siRNA) directed against Notch1. The therapeutic strategy leverages the synergistic effects of mitoxantrone-induced apoptosis or senescence in activated hepatic stellate cells (HSCs) and the silencing of Notch1 signaling, a critical pathway in HSC activation and fibrogenesis. By specifically targeting the activated HSCs, this delivery system aims to reduce the progression of liver fibrosis while minimizing systemic toxicity associated with mitoxantrone.
- Other names
- DVAP-modified exosomes for co-delivery of mitoxantrone and Notch1 siRNADVAP-Exo + MIT + siRNA targeting Notch1
02
Targets
NOTCH (Neurogenic locus notch homolog protein 3)TOP2A (DNA topoisomerase II)
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