Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

The starting dose is 1.5×108 vg, 0.05 mL eye/dose. If drug-related dose-limiting toxicity (DLT) events are observed in \< 2 of the 6 evaluable subjects within 6 weeks after the dosing of NFS-02 at the starting dose, the dose can be escalated to 5.0×108 vg, 0.05 mL eye/dose after the approval by SRC. If drug-related dose-limiting toxicity (DLT) events are observed in \< 2 of the 6 evaluable subjects within 6 weeks after the dosing of NFS-02 at the 5.0×108 vg, 0.05 mL eye/dose, the dose can be escalated to 1.5×109 vg, 0.05 mL eye/dose after the approval by SRC. If drug-related dose-limiting toxicity (DLT) events are observed in ≥ 2 of the 6 evaluable subjects within 6 weeks after the dosing of NFS-02 at the starting dose, the dose can be de-escalated to 5.0×107 vg, 0.05 mL eye/dose after the approval by SRC.