Neurophth’s gene therapy granted orphan drug designation

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For Leber hereditary optic neuropathy associated with an ND4 mutation: NR082 is an ophthalmic AAV-based gene therapy. It uses an AAV2 vector to express the human ND4 gene in the retinal ganglion cells to repair optic neuropathy caused by the mutation. Investigator-initiated trials began in 2011, which demonstrated the long-term safety, effectiveness, and durability of AAV gene therapy in […]