Key clinical point: Factor VIII expression was detected on liver biopsies at more than 2 years after a single infusion of AAV gene therapy for hemophilia A.
Major finding: A dose-dependent increase was seen in the percentage of hepatocytes that stained positive for vector genomes (1.3% in the lower dose and 32% in the higher dose patients).
Study details: A liver biopsy substudy of a phase 1/2 study, with results from the first two patients.
Disclosures: Dr. Fong is an employee of BioMarin Pharmaceuticals.
Fong S et al. 2020 ISTH Congress, Abstract OC 03.2.