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Company Applied Genetic Technologies Corp.
DescriptionAdeno-associated virus (AAV) vector expressing the human cyclic nucleotide gated channel beta 3 (CNGB3) gene
Molecular Target Cyclic nucleotide gated channel beta 3 (CNGB3)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPhase I
Standard Indication Ophthalmic (unspecified)
Indication DetailsTreat achromatopsia
Regulatory Designation U.S. - Orphan Drug (Treat achromatopsia);
EU - Orphan Drug (Treat achromatopsia)
PartnerPfizer Inc.

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