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GenSight Biologics S.A. (GenSight), a clinical-stage biotechnology company discovering and developing novel gene therapies for neurodegenerative retinal diseases, and in the future, of the central nervous system, today announced enrollment of the first patient in both RESCUE and REVERSE, two parallel pivotal Phase III trials with the Company’s lead product candidate GS010 for the treatment of Leber’s Hereditary Optic Neuropathy (LHON).


The pivotal trials are intended to determine whether GS010 can halt or reverse vision loss associated with LHON due to the NADH dehydrogenase 4 (ND4) mutation or be effective as prophylaxis for vision loss in an eye not yet affected. The trials will also seek to identify the therapeutic window of opportunity for treatment after onset of disease.As early intervention is potentially a major factor in maximizing therapeutic success, the two clinical trials will focus on treating patients who have manifested visual decline for up to one year. RESCUE is expected to enroll 36 patients with an onset of vision loss up to 6 months in duration, while REVERSE is expected to enroll 36 patients with an onset of vision loss ranging from 7 to 12 months in duration.