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Liver biopsies show persistent FVIII after gene therapy for hemophilia A

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.