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Company Spark Therapeutics Inc.
DescriptionAdeno-associated virus (AAV) serotype 2 vector expressing the CHM gene, which encodes rab escort protein 1 (CHM; REP1)
Molecular Target Rab escort protein 1 (CHM) (REP1)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPhase I/II
Standard IndicationBlindness
Indication DetailsTreat choroideremia
Regulatory Designation

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