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Biotech Innovations
February 11, 2020

Investigational Hemophilia A Gene Therapy Shows Sustained Benefit

JAMA. 2020;323(6):499. doi:10.1001/jama.2020.0470

An experimental gene therapy for hemophilia A remained effective up to 3 years after a single infusion, researchers recently reported in the New England Journal of Medicine. BioMarin Pharmaceutical’s valoctocogene roxaparvovec encodes factor VIII, the blood-clotting protein that’s missing or low in people with hemophilia A, the most common form of the disease.