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AMT-130

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Company uniQure N.V.
DescriptionAdeno-associated viral serotype 5 (AAV5) vector carrying cassette-optimized synthetic microRNAs to silence the Huntingtin (HTT) gene
Molecular Target
Mechanism of Action 
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationHuntington's disease (HD)
Indication DetailsTreat Huntington's disease (HD)
Regulatory Designation
Partner

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