Log In
Print
BCIQ
Print
Print this Print this
 

AAV2-hRPE65v2

  Manage Alerts
Collapse Summary General Information
Company Spark Therapeutics LLC
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

Partner


 Product Details
 Archive Items are loading loading
Get a free BioCentury trial today