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Early data ‘promising’ for gene therapy for Leber’s hereditary optic neuropathy
GenSight Biologics reported “promising” preliminary data on its gene therapy product for the treatment of neurodegenerative retinal disease, according to a press release.The study included 15 patients with Leber’s hereditary optic neuropathy (LHON) grouped into five groups, with each group of three patients receiving an escalating dose of GS010 via single intravitreal injection in the more severely affected eye. Mean onset of disease was 6 years. Enrollment in the phase 1/2 safety and tolerability study was completed in April 2015.