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Meeting on the Mesa 2021 | Optogenetics in advanced retinitis pigmentosa

Sue Washer, Applied Genetic Technologies Corporation, Alachua, FL, provides a brief overview of the development of an optogenetic adeno-associated virus (AAV)-based gene therapy in combination with a device that stimulates optic nerve cells for patients with advanced stage retinitis pigmentosa (RP). Patients with advanced RP may have no functioning photoreceptors and the gene therapy delivers via intravitreal injection channelrhodopsins to the optic nerve cells. In a promising Phase I/II trial (NCT04278131), an increased visual response and light sensitivity, as well as a favorable safety profile were reported. This interview took place at Meeting on the Mesa 2021.