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Company Applied Genetic Technologies Corp.
DescriptionAdeno-associated virus (AAV) vector expressing the human cyclic nucleotide gated channel alpha 3 (CNGA3) gene
Molecular Target Cyclic nucleotide gated channel alpha 3 (CNGA3)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard Indication Ophthalmic (unspecified)
Indication DetailsTreat achromatopsia
Regulatory Designation EU - Orphan Drug (Treat achromatopsia)

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