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AAV2-hRPE65v2, SPK-RPE65

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Company Spark Therapeutics Inc.
DescriptionAdeno-associated virus (AAV) encoding the retinal pigment epithelium-specific protein 65kDa (RPE65) gene
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 III
Standard IndicationBlindness
Indication DetailsTreat Leber's congenital amaurosis (LCA); Treat Type 2 Leber's congenital amaurosis (LCA2)
Regulatory Designation

U.S. - Breakthrough Therapy (Treat Leber's congenital amaurosis (LCA));
U.S. - Orphan Drug (Treat Leber's congenital amaurosis (LCA));
EU - Orphan Drug (Treat Leber's congenital amaurosis (LCA))


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