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from left to right: Dr. Katie Carlberg,Dr. Beverly Schaefer, Dr. Kanwal Malhi, Dr. Steve Ambrusko

WNYBC hosted the 3rd Hemostasis Thrombosis Research Symposium on May 2 at UB’s Jacobs School of Medicine. This year’s overarching topic spotlighted Gene Therapies for Non-Malignant Hematologic Conditions. Attendees participated virtually and in-person.

The Symposium Keynote Lecture centered on Lessons and Outstanding Questions in Hemophilia Gene Theraby: A primer for AAV gene therapy. Dr. Linsey George, Children’s Hospital of Philadelphia, Assistant Professor of Pediatrics at University of Pennsylvania School of Medicine, Director of Clinic In Vivo Gene Therapy at Children’s Hospital of Philadelphia and a faculty alum of UB’s School of Medicine presented on this cutting-edge treatment option.

Dr. George lead hemophilia A and B gene therapy clinical trials, served as an invited expert for the Food & Drug Administration on hemophilia gene therapy and adeno-associated viral vectors (AAV) gene therapy toxicities, and is a current board member of the American Society of Gene and Cell Therapy. Her lab’s research focus is on mechanisms that regulate factor VIII/VIIIa cofactor function and to exploit the biochemical insights of FVIII function to develop novel gene-based therapies for hemophilia A.

Dr. George presented her work on Lessons and Outstanding Questions in Hemophilia Gene therapy:  A primer for AAV therapy.

Dr. Kanwal Malhi, Clinical Director of Pediatric Bone Marrow Transplantation at Roswell Park & Oishei Children’s Cancer and Blood Disorders Program presented an Overview of Gene Therapy for Sickle Cell disease. Her research interest focuses on providing patients with alternative stem cell donor options and efforts reducing transplant-related complications to improve patient outcomes, and she has clinical expertise in transplants for non-malignant hematologic disorders.

Dr Katie Carlberg is a clinical assistant professor of pediatrics at University at Buffalo and Roswell Park. She is a nonmalignant hematologist who works with WNY BloodCare. She has expertise in the care of children & young adults with hemoglobinopathies, sickle cell disease, thalassemia. Her presentation centered on an Overview of Gene therapy for Thalassemia.

Dr. Steve Ambrusko’s presentation focused on Patient Perspectives on Gene Therapy for Sickle Cell Disease. Dr. Ambrusko is a clinical assistant professor of pediatrics at University at Buffalo and Roswell Park. He is a nonmalignant hematologist, and director of the Sickle Cell & Hemoglobinopathy Center of WNY. He is a skilled hematologist managing patients across the spectrum of hematology, and also serves as the Medical Director for the Multidisciplinary Vascular Anomalies Center at Oishei Children’s hospital.

Jessica Wulf, LMSW, is the Chief Operating Officer and social worker at WNYBC. She is a member of the Hemophilia National Social Work Working group and the Region 1 representative for social workers. She is a recipient of the 2022 Nursing Excellence Fellowship Award through the National Hemophilia Foundation. She rounded out the Symposium event presenting Psychosocial Assessment for Gene Therapy in Hemophilia.

WNY BloodCare is committed to amplifying the work of researchers and clinicians supporting non-malignant hematology to grow interest in the field and treatment options.  WNYBC’s Discovery & Innovation Award provides funding to researchers who meet award criteria. Find information on the Discovery & Innovation Award at https://wnybloodcare.org/discovery-and-innovation-award/.