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Company Voyager Therapeutics Inc.
DescriptionAAV-based gene therapy designed to knock down mutant superoxide dismutase 1 (SOD1)
Molecular Target Superoxide dismutase 1 (SOD1)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationAmyotrophic lateral sclerosis (ALS)
Indication DetailsTreat monogenic amyotrophic lateral sclerosis (ALS)
Regulatory Designation

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