Leber's hereditary optic neuropathy

GenSight Readying to Ask EMA to Approve Gene Therapy for LHON

GenSight Biologics announced it has finished a pre-submission meeting with the European Medicines Agency (EMA), a key step in filing a request for the approval of Lumevoq, its gene therapy for Leber hereditary optic neuropathy (LHON). The company expects to file the request, called a marketing authorization…

Phase 1/2 Study of Gene Therapy to Treat LHON Showing Promise in Those with Early Disease Onset

GenSight Biologics released new preliminary findings from a Phase 1/2 clinical trial assessing the safety and tolerability of the company’s lead investigational gene therapy, GS010, in patients with Leber’s hereditary optic neuropathy (LHON), a maternally inherited mitochondrial disease causing irreversible vision loss. The data show significant improvement for those in earlier stages of the disease. GS010…