Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)

Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children With Retinal Dystrophy Owing to Defects in RPE65 (LCA2)

This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy associated with defects in RPE65.

The follow up study is designed to collect data on longer-term safety and efficacy of AAV2/5-OPTIRPE65 administration in the OPTIRPE65 trial.

Inclusion Criteria:

• Were enrolled and treated in the prior open-label, Phase I/II, dose escalation study involving intraocular administration of AAV2/5-OPTIRPE65

Exclusion Criteria:

• Individuals will be excluded if they are unwilling or unable to meet with the requirements of the study.