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Company Voyager Therapeutics Inc.
DescriptionAdeno-associated virus (AAV)-based gene therapy targeting survival of motor neuron 1 telomeric (SMN1)
Molecular Target Survival of motor neuron 1 telomeric (SMN1)
Mechanism of Action 
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationSpinal muscular atrophy (SMA)
Indication DetailsTreat spinal muscular atrophy (SMA)
Regulatory Designation

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