GenSight’s second LHON gene therapy PhIII stumbles out of the gate — just like the first time
However GenSight’s gene therapy for Leber hereditary optic neuropathy works, it clearly has no significant effect at 48 weeks. But that doesn’t mean the biotech has lost hope in their drug.
The Paris-based biotech reported early Monday that GS010 flopped in their Phase III confirmatory trial at 48 weeks, falling far short of the mark researchers set for an improvement in the sight of the treated eye compared to the sham therapy offered in the other eye. Vision in both eyes deteriorated at almost exactly the same rate.
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