Therapeutically-loaded autologous extracellular vesicles as a lowly-immunogenic gene therapy for the treatment of age-related macular degeneration.

Cells have a language that researchers are only now starting to understand. In order to maintain tissue health, cells communicate by transferring molecular cargo using small cell-to-cell delivery vehicles called extracellular vesicles (EV). The loss of EV communication has been correlated with increased retinal cell death as we age. We believe that replenishing EV and their cargo will restore the communication channels required for maintaining retinal health and slow the progression of retinal degenerations, including age-related macular degeneration (AMD). To investigate this, we will profile EV from healthy and diseased retinas and supplement the degenerating retina with essential retinal EV cargo. We will load and deliver this essential cargo using EV derived from stem cells to reduce the risk of immune attack. This work will identify a therapeutic profile of essential EV cargo required for retinal health, and help develop of EV-based gene therapies for the treatment of AMD.