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Bone marrow mesenchymal stem cell (BMSC)-derived exosomes for the treatment of glaucoma


Glaucoma is one of the world’s leading causes of irreversible blindness. Glaucoma is associated with fluid build-up in the eye resulting in an increased intraocular pressure (IOP). The pressure may cause damage to the optic nerve and lead to progressive degeneration of retinal ganglion cells (RGC) and vision loss. Currently, available treatments for glaucoma delay progression by reducing IOP, but no therapies exist to directly protect RGC from degradation and loss.

Product: Bone marrow-derived mesenchymal stem cells (BMSCs) exosomes confer a significant neuroprotective effect for RGC and prevent vision loss and can be used to treat glaucoma

Competitive Advantage: When compared to potential whole-cell (stem cell) therapies, isolated exosomes have several immediate advantages for clinical translation therapies for glaucoma. Isolation and purification of BMSC‐derived exosomes, or their therapeutically active components, is relatively simple via centrifugation. BMSC‐derived exosomes are stable, and once isolated, can be stored at 4C for months to years. Prior clinical trials of BMSC‐derived exosomes administered systemically have shown a positive safety profile. These exosomes are immunologically inert and, due to their small size and stability, are also easy to dose & deliver and will also readily diffuse from the vitreous into the retinal cell layers. Significant therapeutic- neuroprotective effects for isolated BMSC‐ derived exosomes have been shown in in vitro and in vivo glaucoma models. BMSC‐ derived exosomes are a promising potential cell‐free therapy for glaucoma and degenerative ocular diseases associated with loss of RGC.


Fig: RGC in a 1‐mm region of retina either side of the optic nerve head. Percentage of surviving RGC in comparison to intact controls is given above each group. Asterisks indicate a significant difference from intact, ONC + BMSC Exosomes and ONC + SiScr Exosomes groups (p < .05).

Bone Marrow‐Derived Mesenchymal Stem Cells‐Derived Exosomes Promote Survival of Retinal Ganglion Cells Through miRNA‐Dependent Mechanisms.

Mead B and Tomarev S. Stem Cells Transl Med. 2017 Apr; 6(4): 1273–1285.

Inventors: Ben Mead and Stanislav Tomarev

For licensing: NIH Ref# E-044-2018


Mala Dutta, Ph.D
Office of Translational Research

Last updated: May 18, 2020