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Early data ‘promising’ for gene therapy for Leber’s hereditary optic neuropathy
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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.
At 48 weeks after injection, patients with disease onset of less than 2 years gained an average of 30 letters in the treated eye and 13 letters in the untreated eye, according to the release. In patients with disease onset of more than 2 years, there was no significant difference in letter gain between eyes.
According to the release, the GS010 uses mitochondrial targeting sequence technology and an adeno-associated virus vector to address defects inside mitochondria.