Stuart Orkin, M.D.

Dana-Farber Cancer Institute
Boston Children's Hospital
Howard Hughes Medical Institute
Stuart Orkin photo credit Dana-Farber Cancer Institute
stuart_orkin@dfci.harvard.edu
Stuart Orkin publications
Orkin Laboratory

The Orkin laboratory has been involved in the study of blood disorders for more than 4 decades. Research of the group has examined the development and function of the blood system, the relationship between cancer and stem cells, and the mechanisms responsible for self-renewal of stem cells and the switch from fetal to adult hemoglobin. Prior work from the Orkin laboratory identified BCL11A as the physiological regulator of the fetal-to-adult hemoglobin switch, and demonstrated that gene editing at an erythroid enhancer within the BCL11A gene itself can be used to reactivate fetal hemoglobin expression. These findings were translated into the first approved gene editing therapy for human disease. Currently, the laboratory is focusing its efforts on a deep mechanistic understanding of how fetal globin silencing is controlled, and how it can be reversed by small molecules for treatment of the major hemoglobin disorders, sickle cell disease (SCD) and beta-thalassemia. Multidisciplinary approaches are used including biochemistry, chemical biology, structural studies, and mouse modeling. 

Biosketch

 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. He received a BS from MIT and an MD from Harvard Medical School. He was a USPHS Research Associate in the Laboratory of Molecular Genetics, NICHHD, NIH under the supervision of Dr. Philip Leder from 1973-1975. Following his experience at NIH, Dr. Orkin returned to Boston where he undertook additional training in pediatrics and pediatric hematology/oncology. He served as Chairman of the Department of Pediatric Oncology at the Dana Farber Cancer Institute from 2000-2016. 

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.

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