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.