ND1 mutant Leber Hereditary Optic Atrophy

The purpose of this clinical study is to evaluate Safety, tolerability and preliminary efficacy of NFS-02 ophthalmic injection in the treatment of LHON caused by mitochondrial ND1 gene mutation. This study will enroll subjects aged ≥ 18 years and ≤ 75 years old, and let them receive a single unilateral intravitreal (IVT) injection of NFS-02 to evaluate its safety, tolerability and efficacy. All subjects had clinical manifestations of ND1 mutation-related LHON-induced vision loss, the mutation was detected by the laboratory (CLIA-certified laboratory) as the G3460A site mutation, and the vision loss lasted > 6 months and < 10 years . Currently, there is no effective treatment for LHON subjects. The development of drugs with novel mechanisms of action is considered to be of great importance for future clinical practice. This study can provide a basis for verifying the safety, tolerability and effectiveness of this novel therapy.