UC San Diego Health Joins National Clinical Trial on Hemophilia B Gene Therapy

69-year-old hemophilia sufferer joins trial after lifetime of spontaneous bleeding —

The Hemophilia and Thrombosis Treatment Center at UC San Diego Health has joined a nationwide clinical trial testing a potential gene therapy that may one day provide a better and long-lasting treatment for people with hemophilia B.

The Phase I/II open-label trial is sponsored by Baxalta Incorporated and will assess the safety and optimal dosing level of an investigational blood clotting factor IX gene therapy treatment. … Read the Full Story from the UC San Diego Newsroom


Annette Von Drygalski, MD, PharmDDirecting the Hemophilia and Thrombosis Treatment Center at UC San Diego Health is Annette Von Drygalski, MD, PharmD, Associate Clinical Professor in the Division of Hematology-Oncology, Department of Medicine.

In her research, Dr. Von Drygalski is a basic, translational and clinical scientist specializing in hemostasis and thrombosis and particularly in hemophilia.

She is conducting a number of international clinical research trials on potential new treatment options for patients with hemophilia.