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bone marrow mesenchymal stem cell-derived small extracellular vesicles
- Development stage
- Unknown
- Lead developer
- Mahidol University
- Modality
- Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Ophthalmic
01
Overview
This is an investigational cell-free biologic therapy developed by Mahidol University's Faculty of Medicine Siriraj Hospital for the treatment of retinitis pigmentosa. The therapy consists of Good Manufacturing Practice (GMP)-compliant small extracellular vesicles (sEVs) harvested from bone marrow-derived mesenchymal stem cells (BM-MSCs). Administered via a single intravitreal injection, these vesicles are designed to deliver the therapeutic benefits of MSCs—specifically anti-inflammatory, neuroprotective, and anti-apoptotic effects—without the risks associated with live cell transplantation, such as vitreoretinal traction or immune rejection. The product is currently being evaluated in a Phase 1/2 clinical trial to assess its safety and efficacy in patients with this progressive inherited retinal disease.
- Other names
- BM-MSC-derived sEVsGMP-compliant BM-MSC-derived sEVsMSC-sEVsbone marrow mesenchymal stem cell-derived sEVs
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