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SPK-TPP1

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Company Spark Therapeutics Inc.
DescriptionAdeno-associated virus (AAV) that delivers the tripeptidyl peptidase 1 (TPP1)gene
Molecular Target Tripeptidyl peptidase 1 (TPP1)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard Indication Neurology (unspecified)
Indication DetailsTreat neuronal ceroid lipofuscinosis type 2 (CLN2) disease caused by TPP1 deficiency
Regulatory Designation U.S. - Orphan Drug (Treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease caused by TPP1 deficiency)
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