Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)

An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)

A clinical trial of AAV2/5 vector for patients with Defects in RPE65

A dose escalation and dose expansion (Phase I/II) trial of adults and children with retinal dystrophy associated with defects in RPE65. ATIMP will be administered to one eye only in a single sub-retinal procedure

Key Inclusion Criteria:

• Aged 3 years or older
• Early-onset severe retinal dystrophy consistent with RPE65 deficiency

Key Exclusion Criteria:

• Females who are pregnant or breastfeeding
• Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.