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AT342

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Company Audentes Therapeutics Inc.
DescriptionAdeno-associated viral serotype8 (AAV8) vector carrying a functional version of the UDP glucuronosyltransferase 1 family polypeptide A1 (UGT1A1) gene
Molecular Target UDP glucuronosyltransferase 1 family polypeptide A1 (UGT1A1)
Mechanism of Action 
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard Indication Metabolic (unspecified)
Indication DetailsTreat Crigler-Najjar syndrome Type I
Regulatory Designation
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