Drug intelligence / Profile preview

EXOB-001

Development stage
Phase 2
Lead developer
EXO Biologics
Modality
Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intratracheal
01

Overview

EXOB-001 is an investigational exosome-based therapy consisting of extracellular vesicles (EVs) derived from cultured human allogeneic umbilical cord mesenchymal stromal cells (hMSCs). It is being developed for the prevention of bronchopulmonary dysplasia (BPD) in preterm newborns. The drug is administered via intratracheal instillation, delivering EVs directly into the lungs. These vesicles are believed to reduce inflammation and promote tissue repair and regeneration by modulating immune responses, potentially preventing or reducing the severity of BPD. EXOB-001 has received Orphan Drug Designation from both the FDA and EMA for this indication and is currently undergoing Phase 1/2 clinical trials in extremely premature infants[1][2][3][4][5][8].

Other names
Allogeneic umbilical cord mesenchymal stromal cell-derived extracellular vesiclesMSC-derived exosomesUmbilical cord mesenchymal cells-derived extracellular vesicles
02

Targets

TNFA (Tumor necrosis factor alpha (soluble))TGFB (GARP–latent transforming growth factor beta 1 complex)VEGFA (Vascular endothelial growth factor A)IL-6 (Interleukin 6)

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