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Company Horama S.A.S.
DescriptionSerotype 4 rAAV that delivers the gene for RPE65
Molecular Target Retinal pigment epithelium-specific protein 65kDa (RPE65)
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)
Regulatory Designation EU - Orphan Drug (Treat Leber's congenital amaurosis (LCA))

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