Key clinical point: SPK-8011 gene therapy for hemophilia A provides stable and durable factor VIII expression with no major safety concerns.
Major finding: FVIII activity was less than 2% before treatment and 5.2%-19.8% at 2 to 3.3 years after treatment.
Study details: A phase 1/2 study of 14 patients.
Disclosures: Spark Therapeutics sponsored the SPK-8011 study. Dr. George disclosed consulting and/or data safety monitoring board activity for Pfizer and AvroBio.
George L et al. 2020 ISTH Congress, Abstract OC 03.5.