CHOP HTC Receives Research Grant from Brandywine Valley Hemophilia Foundation

by Karen Bowe-Hause, Director of Member and Community Relations

The Children’s Hospital of Philadelphia (CHOP) Hemophilia Treatment Center was the recent recipient of a research grant from Brandywine Valley Hemophilia Foundation, in memory of Nancy Haldeman Ramsey Olson, R.N. Nancy (1935-2025) was a mother, nurse, and hero in the Bleeding Disorder Community. She was the founding member of the Brandywine Valley Hemophilia Foundation and a pioneer in home treatment training at CHOP to improve the lives of patients with hemophilia in the 1960s.

Denise Sabantino, PhD, Research Associate Professor of Pediatrics at CHOP provided the following information on the research being done by the HTC team and the importance of this new grant:

CHOP has a longstanding commitment to developing gene therapy for hemophilia. For over 25 years, under the leadership of Dr. Katherine High, investigators at CHOP have been developing gene-based therapies with the goal of creating a single treatment that could last a patient’s lifetime. The goal is to achieve continuous levels of clotting factor (factor IX or factor VIII) in the blood, ensuring it is always present at the time of a bleeding episode. In the last few years, several gene therapy products have been approved for both hemophilia B and for hemophilia A. For some patients, this treatment has been truly life changing because they no longer require (prophylactic) treatment with protein products. However, some patients have returned to treatment with their protein replacement therapy.

Dr. Sabantino says her goal is to develop the next generation of gene therapy products for hemophilia A.The donation from the Brandywine Valley Hemophilia Foundation to our research in honor of Nancy Olson has been critical to sustaining our research efforts especially in these times when our NIH funding has been delayed.

“My laboratory at CHOP focuses on studying hemophilia A and developing a gene therapy for hemophilia A. Our research focuses on studying how gene therapy works and developing strategies to improve these gene therapy approaches,” said Dr. Sabantino. The areas of research in her laboratory include: the development of new FVIII protein variants that can boost the function of the protein and incorporation of these proteins into gene therapy approaches; targeting the new gene to the specific cells in the liver that naturally produce FVIII and to learn if the FVIII produced in those specific liver cells will overcome some of the challenges of gene therapy for hemophilia A; developing a different gene therapy approach by correcting the gene rather than inserting a new gene; and understanding how liver health will impact gene therapy.