Study Status: NOT_YET_RECRUITING
Recruit Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Official Title: A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects With RPE65-associated Leber Co
Brief Summary: The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.