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Also known as: DTX101 AAVrh10 FIX

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Company Dimension Therapeutics Inc.
DescriptionRecombinant adeno-associated virus (AAV) of serotype 10 (AAVrh10) vector delivering the wild-type Factor IX gene
Molecular Target Factor IX
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPhase I/II
Standard IndicationHemophilia
Indication DetailsTreat hemophilia B; Treat moderate/severe to severe hemophilia B
Regulatory Designation U.S. - Fast Track (Treat hemophilia B);
U.S. - Orphan Drug (Treat hemophilia B)
PartnerRegenxBio Inc.

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