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Company uniQure N.V.
DescriptionRecombinant adeno-associated viral (AAV) serotype 5 (AAV5) vector carrying glial cell-derived neurotrophic factor (GDNF) gene
Molecular Target Glial cell-derived neurotrophic factor (GDNF)
Mechanism of Action 
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationAmyotrophic lateral sclerosis (ALS)
Indication DetailsTreat amyotrophic lateral sclerosis (ALS)
Regulatory Designation
PartnerTreeway B.V.

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