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Valoctocogene Roxaparvovec

CHEMBL4297579 Phase 4 Approuvé Gene
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

Valoctocogene roxaparvovec is an adeno-associated virus (AAV5) gene therapy vector that delivers a functional copy of the B-domain deleted factor VIII gene to hepatocytes, enabling the liver to produce sufficient factor VIII to prevent bleeding in adults with severe hemophilia A. Following a single intravenous infusion, transduced hepatocytes continuously express factor VIII, potentially eliminating the need for regular prophylactic factor VIII infusions. It represents a potentially curative approach for hemophilia A.

Mécanisme d'action

Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

Mécanisme

Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.

HBD / HBA

- / -

No targets recorded

Target interaction data is not yet available for this drug.

No interactions recorded

Drug interaction data is not yet available for this compound.

No side effects recorded

Side effect data is not yet available for this drug.

Foire aux questions

Valoctocogene roxaparvovec is an adeno-associated virus (AAV5) gene therapy vector that delivers a functional copy of the B-domain deleted factor VIII gene to hepatocytes, enabling the liver to produce sufficient factor VIII to prevent bleeding in adults with severe hemophilia A. Following a single intravenous infusion, transduced hepatocytes continuously express factor VIII, potentially eliminating the need for regular prophylactic factor VIII infusions. It represents a potentially curative approach for hemophilia A.

Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.

Yes, Valoctocogene Roxaparvovec is an approved drug. It has reached clinical phase 4. It is classified as a Gene.

{# References & Data Sources section for drug detail pages. Renders standard pharmacological database links plus the drug's data_sources field. #}

References & Data Sources

  • ChEMBL — European Bioinformatics Institute (EBI). CHEMBL4297579. Open-access bioactivity database.

Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.

Avertissement médical

This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.

Data sources: ChEMBL, PubChem, DailyMed.