GenSight Biologics � GS011 LHON-ND4

GenSight, a London-based biotech company, is a gene therapy company seeking to apply pathbreaking science to help patients with severe inherited diseases of the eye and central nervous system. GenSight, has two lead product candidates for the treatment of sight-threatening retinal degenerative diseases, together with products in preclinical development targeting ophthalmic and neurodegenerative diseases. LHON-ND4 (EU) is a drug for the treatment of Leber Hereditary Optic Neuropathy. The ND4 gene is normally located in the mitochondria where ND4 proteins are synthesized. GS010 allows allotopic expression of the mitochondrial gene ND4 in the nucleus thanks to a proprietary Mitochondrial Targeting Sequence that shuttles the messenger RNA from the nucleus directly to the outer membrane of the mitochondria. There, the ND4 proteins are synthesized and incorporated into the mitochondria. Wild-type ND4 proteins then integrate into Complex I of the respiratory chain and rescue the deficiency. ClinicalTrials.gov Identifier: NCT03293524