Stuart H. Orkin, MD

Dr. Stuart H. Orkin is the David G. Nathan Distinguished Professor of Pediatrics at Harvard Medical School and an HHMI Investigator at Boston Children’s Hospital. Orkin has defined the molecular basis of human blood disorders and mechanisms governing blood cell development. Previously he served as Chairman of the Department of Pediatric Oncology at the Dana Farber Cancer Institute from 2000-2016. He received a BS from MIT and an MD from Harvard Medical School. Dr. Orkin served on the National Research Council Committee on Mapping the Human Genome and as co-chair of the Panel to Assess the NIH Investment in Gene Therapy.

He provided the first comprehensive molecular dissection of an inherited disorder (the thalassemia syndromes), and characterized genes responsible for other human blood disorders, including X-linked chronic granulomatous disease (the first positional cloning). Orkin identified the first hematopoietic transcription factors (the GATA family) and characterized their roles in blood cell development and cancer. His studies of BCL11A, a repressor of fetal hemoglobin (HbF), have illuminated regulation of globin gene switching and led to approved gene editing therapy for both sickle cell disease and beta-thalassemia.

Dr. Orkin was elected to the National Academy of Sciences (NAS), National Academy of Medicine (NAM), American Academy of Arts and Sciences, and the American Philosophical Society, His honors and awards include the E. Mead Johnson Award, Warren Alpert Prize, Helmut Horten Foundation Prize, Distinguished Research Award from the Association of American Medical Colleges (AAMC), E. Donnall Thomas, Dameshek and Basic Science Mentor Awards of the American Society of Hematology (ASH), Jessie the Stevenson Kovalenko Medal of the National Academy of Sciences (2013), William A. Allan Award of the American Society of Human Genetics (2014), George M. Kober Medal of the American Association of Physicians (2018), Mechthild Esser Nemmers Prize in Medical Science of Northwestern University (2018), King Faisal Prize in Medicine (2020), Harrington Prize for Innovation in Medicine (2020), Tobias Prize Lecture of the International Society of Stem Cell Research (2021), Gruber Prize in Genetics (2021), Canadian Gairdner International Award (2022), Society of Memorial Sloan Kettering Prize (2023), George Stamatoyannopoulos Mentorship Award of the American Society of Gene and Cell Therapy (2023), Honorary Doctorate (PhD honoris causa) University of Montreal, and the Elaine Redding Brinster Prize (2024). He was named to the Time100 2024 list of the 100 most influential individuals and the 20204 Time100 Health List. Most recently, he was awarded the 2024 Shaw Prize in Life Science and Medicine.