Clinical Trial Update: Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations [Phase 1]

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Summary

A Phase 1 clinical trial update for a gene therapy targeting Leber Congenital Amaurosis caused by RPE65 mutations; the study status is completed.

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Diseases / indications

• Leber Congenital Amaurosis

Drugs mentioned

• AAV2-hRPE65 gene therapy (AAV2-hRPE65)

Snippet

Sponsor: Hadassah Medical Organization | Conditions: Leber Congenital Amaurosis | Phase 1 | Status: COMPLETED