Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year

Inclusion Criteria

• •Main Selection Criteria:
• •Age 15 years or older on the date of signed informed consent.
• •Clinically manifested vision loss due to ND4 LHON, to any extent, in at least one eye.
• •Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion Visit (Visit 2).
• •Main Non-Selection Criteria:
• •Contraindication to intravitreal injection in any eye.
• •Subjects refusing to discontinue idebenone.
• •Previous vitrectomy in either eye.
• •Narrow angle in any eye contra-indicating pupillary dilation.
• •Presence of known/documented mutations, other than the G11778A ND4 LHON-causing mutation, which are known to cause pathology of the optic nerve, retina or afferent visual system.
• •History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation.
• •Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion Visit (Visit 2).
• •Each eye of the subject must maintain at least Hand Motion (HM) visual acuity, as defined by the study's SOP for visual acuity testing.
• •Documented results of genotyping showing the presence of the G11778A mutation in the ND4 gene and the absence of the other primary LHON-associated mutations (ND1 or ND6) in the subject's mitochondrial DNA.