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rAAV2/2_ND4 (GS010)

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Company GenSight Biologics S.A.
DescriptionGene therapy product containing the human ND4 gene
Molecular Target NADH dehydrogenase subunit 4 (ND4)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPhase III
Standard IndicationOptic neuropathy
Indication DetailsTreat Leber's hereditary optic neuropathy (LHON)
Regulatory Designation

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