Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients

Inclusion criteria

• ✓. Aged 15 years or older at the time of vision loss onset
• ✓. Clinically manifested vision loss due to ND4 LHON in both eyes
• ✓. BCVA of at least LogMAR +2.39, measured at the FrACT scale, in both eyes
• ✓. Documented results of genotyping showing the presence of pathogenic LHON-associated mutation(s) in the ND4 mitochondrial gene and the absence of other pathogenic LHON-associated mutations in the patient's mitochondrial DNA; absence of pathogenic mutations, other than the ND4 LHON-causing mutation(s), which are known to cause pathology of the optic nerve, retina or afferent visual system. Historic results of genetic analysis are acceptable upon Sponsor approval.
• ✓. Vision loss duration from 6 months to 1.5 years in the first affected eye at the Inclusion visit (Day 1)
• ✓. No limitations to OCT image collection that would prevent high quality, reliable images from being obtained in both eyes, as determined by the reading center
• ✓. Clear ocular media and adequate pupillary dilation to permit thorough ocular examination, as assessed by the Investigator
• ✓. Human immunodeficiency virus (HIV) negative serology