Drug intelligence / Profile preview

human umbilical cord mesenchymal stem cell-derived exosomes

Development stage
Phase 2
Lead developer
Xijing Hospital
Modality
Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous, Intranasal, Ophthalmic, Intralesional, Inhalation, Parenteral
01

Overview

Human umbilical cord mesenchymal stem cell-derived exosomes (hUC-MSC-Exos) are nanosized extracellular vesicles, typically ranging from 30 to 200 nm in diameter, secreted by mesenchymal stem cells harvested from human umbilical cord tissue. As a cell-free therapeutic platform, these exosomes carry a complex cargo of bioactive molecules, including microRNAs (e.g., miR-129-5p, miR-19a), long non-coding RNAs (e.g., lncRNA-9969), and proteins (e.g., TGF-beta 1), which mediate intercellular communication and tissue regeneration. The therapeutic mechanism involves the modulation of multiple signaling pathways, such as the activation of the SIRT1-FXR axis to inhibit NLRP3 inflammasome activity in macrophages and the targeting of ACSL4 to suppress ferroptosis and lipid peroxidation. hUC-MSC-Exos are currently being investigated in numerous clinical trials for diverse indications, including inflammatory bowel disease, autoimmune encephalitis, vitiligo, dry eye disease, and neurodegenerative disorders, offering a potential alternative to stem cell transplantation with reduced risks of immunogenicity and tumorigenicity.

Other names
脐带间充质干细胞外泌体human umbilical cord mesenchymal stem cell-derived exosomehUC-MSC-derived exosomesumbilical cord MSC-derived exosomes
02

Targets

TGFB1 (Transforming growth factor Beta-1 proprotein)ENG (Integrin Beta 6)SIRT1 (NAD-dependent protein deacetylase sirtuin-1)NLRP3 (Nod-like receptor family pyrin domain-containing protein 3)

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