ND1 mutant Leber Hereditary Optic Atrophy

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