GenSight Biologics

GenSight Biologics is a clinical-stage biotechnology company with a focus on discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. Leveraging the Mitochondrial Targeting Sequence (MTS) and optogenetics, the company's pipeline aims to preserve or restore vision in patients with severe degenerative retinal diseases. Its lead product candidate, GS010, is in Phase III trials in Leber’s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease causing irreversible low vision and legal blindness in teens and young adults. With a founding year of 2012 and headquartered in France, GenSight Biologics received a significant €9.28M Post-IPO Equity investment on May 7, 2024. The company's gene therapy-based approach involves administering product candidates via a single treatment to each eye through intravitreal injection to provide patients with sustainable functional visual recovery. This aligns with the company's slogan of applying pathbreaking science to transform the lives of patients with severe retinal neurodegenerative diseases.