Gene therapy: retinal & CNS degenerative pathologies and mitochondrial disease. Lead, GS010, Ph 3 for Leber Hereditary Optic Neuropathy (LHON). REVERSE Ph 3 topline data announced Apr 2018, RESCUE Ph 3 data exp 1Q 2019. Third Ph 3, REFLECT, exp 2020. Filing for MAA exp Q4 2019 in EU and 2020 in US. Pipeline concomitant program, GS030: optogenetic combo drug + enhancing goggle device entering clinic 4Q18: non-syndromic retinitis pigmentosa. Optogenetic program and targeted disease immediately transferable to dry AMD. Platform combines mono-genetic therapy-based approach w core technology platform and proprietary Mitochondrial Targeting Sequence. Optogenetic program transduces retinal ganglion cells into light responsive cells, bypassing retinal degenerations that destroy photoreceptors.
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