Why Choose the Novel Therapeutics for Bleeding Disorders (NoT Bleeding) Program

The NoT Bleeding Program at Children's Hospital of Philadelphia (CHOP) is co-led by a team of world-class experts who together have more than two decades of clinical experience in gene therapy for hemophilia and in innovating care for patients with bleeding disorders. Basic, translational and clinical research efforts led by CHOP investigators over the past three decades have resulted in critical developments allowing the realization of hemophilia gene therapy in the clinic.

Four of the NoT Bleeding investigators have their own fully staffed laboratories with a longstanding history of conducting basic science and translational research. Experts from the Clinical In Vivo Gene Therapy research team support the program’s clinical research.

Principal Investigators

Lindsey George, MD, is a physician-scientist internationally known for her leadership in hemophilia A and B gene therapy clinical trials and has served as an invited expert for the U.S. Food & Drug Administration on hemophilia gene therapy and adeno-associated viral vectors (AAV) gene therapy toxicities. She additionally runs a basic science research laboratory focused on coagulation factor VIII, developing novel gene therapy approaches for hemophilia A and investigating translational questions that have emerged from AAV clinical trials.

Rodney Camire, PhD, holds the Children’s Hospital of Philadelphia’s Endowed Chair in Hematology Research. He is an international expert on the biology of hemostasis. His laboratory focuses on the molecular and cellular processes that contribute to and regulate blood coagulation as well as therapeutic drug development for bleeding and clotting disorders.

Benjamin Samelson-Jones, MD, PhD, is nationally recognized for characterizing and identifying hyperactive gene variants for hemophilia gene therapy. He has been a clinical investigator for gene therapy clinical trials for more than seven years. His current research is focused on gene therapy, immune responses related to hemophilia, and repurposing therapies for novel indications.

Leslie Raffini, MD, MSCE, is the Medical Director of CHOP’s Hemostasis and Thrombosis Center. She is a clinical investigator whose research is focused on improving the care for patients with bleeding and clotting disorders. She is nationally recognized for her leadership in developing the hemostasis and thrombosis program at CHOP and serves on several national committees to help guide the care of patients with bleeding disorders.

Bhavya Doshi, MD, is a physician-scientist with expertise in hemostasis and immunology. Her laboratory focuses on identifying novel therapies for factor VIII inhibitors and other immune-mediated disorders. She is also an Assistant Director of the CHOP Clinical Coagulation Laboratory and focuses on coagulation assay development.

Other NoT Bleeding Team Members

Hilary Whitworth, MD, MSCE, is an attending hematologist and clinical researcher specializing in hemostasis and thrombosis. She is a National Hemophilia Foundation Clinical Fellow and completed the CHOP Cell and Gene Therapy Clinical Training Course.

Lauren Rosenblum, MSN, RN, is the nurse navigator for the NoT Bleeding Program. She has 18 years of experience in providing direct, in-patient care. She is developing expertise in hemophilia, bleeding disorders and gene therapy.

Danielle Deery, JD, MURP, LSSGB, is the administrative manager for the NoT Bleeding Program and provides support for all clinical and administrative activities. She has been at CHOP for more than 10 years and has experience in both the Research Institute and in hospital operations.