Navigating the Evolving Landscape of AAV Gene Therapy for Inherited Retinal Diseases

Navigating the Evolving Landscape of AAV Gene Therapy for Inherited Retinal Diseases

For individuals and families living with inherited retinal diseases (IRDs), gene therapy using adeno-associated viruses (AAVs) has long represented a beacon of hope for preserving or even restoring vision. As a community, we've celebrated milestones like the FDA approval of Luxturna for RPE65-mediated retinal dystrophy, which demonstrated the potential of this technology. However, recent developments suggest that AAV gene therapy is at a critical juncture, facing both significant challenges and ongoing innovation that will shape its future for IRD treatments.

Gene therapy aims to address the root cause of IRDs by delivering functional copies of faulty genes directly to retinal cells, often before significant vision loss occurs. AAVs are commonly used as