Stem Cell-Derived Extracellular Vesicle Drugs to Enter Clinical Exploration in Nine Major Disease Areas

In recent years, extracellular vesicles derived from stem cells have emerged as a hot research topic in regenerative medicine, attracting global attention for their therapeutic potential. With the advancement of the key research program "Stem Cell Research and Organ Repair," the development of stem cell-derived extracellular vesicle drugs has entered a new stage.

This program explicitly prioritizes the large-scale production, quality assessment, and clinical exploration of stem cell derivatives, focusing on conditions such as acute liver failure, stroke, and wound healing. The aim is to overcome the critical bottlenecks preventing extracellular vesicle drugs from transitioning from laboratory research to the market.

Tiny Molecular Factories Carrying Hope for Treatment

Mesenchymal stem cell-derived exosomes are small vesicles secreted by cells, ranging from 30 to 150 nanometers in diameter. These nanoscale vesicles function as "delivery trucks," precisely transporting bioactive substances such as proteins, nucleic acids, and lipids.

More importantly, compared to traditional stem cell therapies, extracellular vesicles offer significant advantages in safety, ease of storage, and standardized production.

Global Research Progress and Breakthroughs

According to ClinicalTrials.gov, over a hundred clinical studies on extracellular vesicles are currently underway worldwide, covering diseases such as liver disease, neurodegenerative disorders, cancer, and rare diseases.

For instance, U.S.-based Direct Biologics is conducting a Phase III clinical trial for its extracellular vesicle drug ExoFlo, targeting acute respiratory distress syndrome (ARDS).

However, challenges remain in evaluating efficacy, achieving large-scale production, and establishing regulatory standards. The field is still in the "drug development exploration" stage. In animal models of acute liver failure and stroke, extracellular vesicles derived from adipose stem cells have demonstrated significant tissue repair and anti-inflammatory effects.