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Early results with ‘optimized’ gene therapy in hemophilia A

Key clinical point: An investigational gene therapy, BAY 2599023, can increase factor VIII levels in patients with hemophilia A.

Major finding: Three of four patients achieved clinically meaningful factor VIII levels, and all were able to stop prophylaxis at some point. However, one patient had to resume prophylaxis, and all four have experienced bleeds on study.

Study details: A phase 1/2, dose-escalation study of four patients with severe hemophilia A.

Disclosures: The trial is sponsored by Bayer in collaboration with Ultragenyx Pharmaceutical Inc. Dr. Pipe disclosed relationships with Bayer and other companies.


Pipe SW et al. EAHAD 2020. Abstract P190.