Abstract

Extracellular Vesicles from Basic Biology to Next Generation Therapy: A Translational Perspective

Ahmed Ibrahim, Research Scientist, Capricor Therapeutics

It is widely accepted in the cell therapy field that most of the therapeutic effects observed in reparative cell therapies using non engrafting cells are due to the paracrine factors secreted by the delivered cells very early after administration. Among these, extracellular vesicles (EV) seem able to recapitulate most of the beneficial responses triggered after cell administration and present important advantages in terms of manufacturing, administration and immunological response. Capricor is now developing an advanced therapeutic based on EVs obtained from previously developed product allogeneic cardiosphere-derived cells (CDC). A manufacturing process is now being developed but initial EV characterization shows we are able to produce equivalent EV batches from different donors. In vitro potency assays suggest that EV, similar to the CDC, are able to modulate the immune response, promote proliferation and induce a regenerative response. In addition, pre-clinical studies demonstrate the low immunogenic profile of the EV obtained from CDC. This will allow the production of off-the-shelf bio-equivalent products that can be delivered into allogeneic patients without producing relevant immune response (no match between donor and patient will be required) and opens the possibility to repetitive treatments. We are studying the ability of the CDC derived EV for promoting tissue regeneration in different disease models. We have observed very promising results in animal where acute damage was induced and we are also testing the ability of this advanced medicinal product in the treatment of chronic diseases.