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This investigational biologic therapy consists of exosomes derived from human umbilical cord mesenchymal stem cells (UC-MSCs), developed by the Zhongshan Ophthalmic Center at Sun Yat-sen University. It is specifically being developed as a cell-free therapeutic approach for treating dry eye syndrome in patients with chronic graft-versus-host disease (cGVHD). Administered as topical eye drops, these nanosized extracellular vesicles contain a cargo of bioactive molecules, including proteins and microRNAs such as miR-204. The therapy exerts immunomodulatory and anti-inflammatory effects by reprogramming proinflammatory M1 macrophages into immunosuppressive M2 macrophages. This transition is achieved through the miR-204-mediated targeting and inhibition of the IL-6/IL-6R/Stat3 signaling pathway, which helps alleviate ocular surface inflammation and improve clinical symptoms of dry eye.
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