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Company RegenxBio Inc.
DescriptionAdeno-associated virus serotype 8 (AAV8) vector delivering the low-density lipoprotein receptor (LDLR) gene
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentIND
Standard IndicationHypercholesterolemia
Indication DetailsTreat homozygous familial hypercholesterolemia (HoFH)
Regulatory Designation

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