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Company Applied Genetic Technologies Corp.
DescriptionAdeno-associated virus serotype 2 (AAV2) vector expressing RPE65
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPhase I/II
Standard IndicationBlindness
Indication DetailsTreat Leber's congenital amaurosis (LCA) or severe early childhood-onset retinal degeneration
Regulatory Designation

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