Clinical Edge

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Gene Therapy for Hemophilia A Associated With Sustained Reductions in Bleeding Rates

Key clinical point: Gene therapy for hemophilia A appears to be associated with sustained reductions in bleeding rates.

Major finding: Bleeding rates were reduced by 96% in the 6 x 1013 vg/kg group by year 3 with the gene therapy.

Study details: Phase 1/2 trial in 15 adults with hemophilia A who received valoctocogene roxaparvovec.

Disclosures: The study was sponsored by manufacturer BioMarin Pharmaceutical. Dr. Pasi reported financial relationships with the study sponsor and other companies.


Pasi KJ et al. 2019 ISTH Congress, Abstract LB 01.2.