GenSight Biologics S.A

GenSight Biologics S.A., a clinical-stage biotechnology company, discovers, develops, and commercializes therapies for mitochondrial and neurodegenerative retinal diseases of the eye and central nervous system. The company develops its products through gene therapy-based mitochondrial targeting sequence and optogenetics technology platforms. Its lead product candidates include LUMEVOQ (GS010), a recombinant AAV2-based gene therapy, which is in Phase III clinical trials for the treatment of leber hereditary optic neuropathy caused by a mutated ND4 gene; and GS030, which is in Phase I/II clinical trial for the treatment of retinitis pigmentosa, as well as in preclinical stage to treat dry age-related macular degeneration. The company is also developing products that are in preclinical stage targeting ophthalmic and neurodegenerative diseases. It has license agreements with Sorbonne Université, CNRS, Inserm and SATT Lutech; Inserm Transfert S.A, Genethon, and Harvard; Massachusetts Institute of Technology; Novartis Pharma AG; Sorbonne University, Centre National de la Recherche Scientifique, Institut National de la Santé et de la Recherche Médicale and Satt Lutech; Association Française contre les Myopathies, Inserm Transfert S.A; and non-exclusive license agreement with Adverum Biotechnologies. The company was incorporated in 2012 and is headquartered in Paris, France.