Overview

A Safety and Efficacy Study of HG004 in Subjects With Leber Congenital Amaurosis

Status:
Not yet recruiting
Trial end date:
2025-12-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
HuidaGene Therapeutics Co., Ltd.
Collaborator:
Cholgene Therapeutics, Inc.