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AAV2-hRPE65v2, voretigene neparvovec (formerly 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)

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Disease CategoryStandard IndicationIndication DetailsPartnersMilestonesLatest Stage of DevelopmentRegulatory Designation
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Total Number of DealsTotal Deal ValueTotal Upfront CashTotal Milestone Payments

1

$50.0M

$50.0M

0


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