GenSight Biologics

GenSight Biologics is a clinical-stage biotechnology company focused on discovering and developing novel therapies for neurodegenerative retinal diseases and central nervous system disorders. GenSight Biologics' pipeline leverages two core technology platforms, Mitochondrial Targeting Sequence (MTS) and Optogenetics in Retinitis Pigmentosa, to help patients with severe degenerative retinal diseases maintain or restore vision. The company's lead product candidate, GS010, is currently in Phase III trials for Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that causes irreversible low vision and legal blindness in adolescents and young adults. Using its gene therapy-based approach, GenSight Biologics’ candidates are designed to provide sustained functional vision recovery through a single treatment per eye via intravitreal injection.