David A. Williams

David A. Williams, MD, is the Leland Fikes Professor of Pediatrics at Harvard Medical School and Chief of Hematology/Oncology at Boston Children's Hospital. A pioneer in gene therapy for blood disorders, his research focuses on hematopoietic stem cell biology and developing genetic treatments for sickle cell disease, immunodeficiencies, and other genetic blood disorders.

Williams leads clinical trials applying lentiviral gene therapy for Wiskott-Aldrich syndrome, sickle cell disease, and cerebral adrenoleukodystrophy. His laboratory investigates Rho GTPase regulation of stem cell functions and develops improved gene transfer methods, recently demonstrating reduced genotoxicity with culture-free editing techniques. Williams' innovations include the shmiR vector system that induces fetal hemoglobin by knocking down BCL11A in sickle cell patients.

Honored with the 2023 ASGCT Founder's Award, Williams previously directed research at the Howard Hughes Medical Institute. His work has produced FDA-approved therapeutics including the thrombopoietin agonist eltrombopag, now being evaluated in pediatric aplastic anemia trials.