Browse Studies — Arctuva

Phase 1

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 Congenital Amaurosis Type 2 (LCA2)

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 mutation…

Leber Congenital AmaurosisInherited Retinal Diseases Caused by RPE65 Mutations

Estimated

~$10 – $8,750

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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 Congenital Amaurosis Type 2 (LCA2)

Optimizing Low Vision Rehabilitation in Emotionally Distressed Patients With Inherited Retinal Diseases