Gensight Biologics SA

GenSight Biologics S.A. is a clinical-stage biotechnology company that discovers, develops, and commercializes novel therapies for neurodegenerative retinal diseases and central nervous system disorders. Its products are based on gene therapy mitochondrial targeting sequence and optogenetics technology platforms. Lead candidates include LUMEVOQ, a recombinant AAV2-based gene therapy in Phase III trials for Leber hereditary optic neuropathy caused by a mutated ND4 gene, and GS030, in Phase I/II trials for retinitis pigmentosa and dry age-related macular degeneration. The company also has preclinical programs in ophthalmic and neurodegenerative diseases, and license agreements with institutions such as Sorbonne Université, CNRS, Inserm, Genethon, Harvard, MIT, and Novartis Pharma AG, as well as a non-exclusive agreement with Adverum Biotechnologies. Incorporated in 2012, it is headquartered in Paris, France.