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valoctocogene roxaparvovec
- Development stage
- Approved
- Lead developer
- BioMarin
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
Valoctocogene roxaparvovec is a gene therapy indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency). It is an adeno-associated virus serotype 5 (AAV5) based gene therapy vector that delivers a functional copy of the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ) to hepatocytes using a liver-specific promoter. This enables the liver to produce and secrete active factor VIII, thereby increasing circulating levels and reducing or preventing bleeding episodes. The therapy is administered as a one-time intravenous infusion and offers long-term production of factor VIII, potentially normalizing bleeding risk in patients with hemophilia A[1][3][4][5][6][8].
- Brand names
- Roctavian
- Other names
- valoctocogene roxaparvovec-rvox
02
Targets
AAVR (AAV receptor)
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