Clinical Edge

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SPK-8011 AAV-mediated Hemophilia A therapy shows stability, durability

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.