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Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A.

Hemophilia A is a genetic disease caused by the deficiency of clotting factor VIII. It is the most common type of hemophilia and occurs much more frequently in males; incidence is estimated at 1 in 4,000-5,000 male births.

People born with hemophilia produce little or no clotting factors. The two main types of hemophilia are A and B. People with hemophilia A are missing or have low levels of clotting factor VIII. People with hemophilia B are missing or have low levels of clotting factor IX. These proteins work with platelets in the clotting process. When blood vessels are injured, clotting factors help platelets stick together to plug cuts and breaks on the vessels and stop bleeding. Many patients with hemophilia experience spontaneous bleeding events that result in progressive and debilitating joint damage.

The current standard of care for most hemophilia A patients who are severely affected today is a prophylactic regimen of intravenous infusions three times per week. Even with prophylactic regimens, many patients still experience spontaneous bleeding events.

Gene Therapy Research and Hemophilia A


Molecule
Condition
Phase (Preclinical, Phase 1, Phase 2, Phase 3, BLA/NDA/MAA, Approval)
  • Valoctocogene Roxaparvovec (BMN 270) AAV-Factor VIII
    Severe Hemophilia A
    MAA
    Target Indication

    Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A.

    Learn More about Valoctocogene Roxaparvovec

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