The Foundation for the National Institutes of Health (FNIH) has awarded its seventh annual Trailblazer Prize for Clinician-Scientists to Vijay G. Sankaran, MD, PhD, of Boston Children’s Hospital and Harvard Medical School. Dr. Sankaran and his team have made significant advances in the understanding of human blood cell development in health and diseases, including sickle cell anemia, cancer, and rare blood disorders.

“Dr. Sankaran’s pioneering studies in genetics and hematology paved the way for a novel sickle cell disease treatment involving the CRISPR gene-editing technology, becoming the first gene therapy approved by the Food and Drug Administration,” said Julie Gerberding, MD, MPH, President and CEO of the FNIH. “This approach can serve as a blueprint for the development of other gene editing therapies and represents the innovative thinking that’s a hallmark of Trailblazer Prize recipients.”

The FNIH’s Trailblazer Prize recognizes the outstanding contributions of early career clinician-scientists whose research translates basic discoveries into novel approaches for diagnosing, preventing, treating, or curing disease and disability.

Dr. Sankaran’s work has focused on the genes and mechanisms underpinning blood cell development and disorders. He discovered the function of a particular gene involved in blood production that, when suppressed using the CRISPR gene-editing technology or a technique known as RNA interference, could treat sickle cell anemia, a painful blood disorder. He identified thousands of gene variants involved in multiple diseases, including blood cancers, and described the role of individual mutations in Diamond-Blackfan anemia, a life-threatening blood disorder. Additionally, Dr. Sankaran’s lab devised a method that can triple the production of red blood cells, which could lead to better, cheaper methods of manufacturing these cells for transfusion.

“I am deeply honored to receive the 2024 FNIH Trailblazer Prize, which recognizes the tremendous work of my laboratory,” Dr. Sankaran said. “Our research uses insights from human genetics to gain a deeper understanding of blood cell production in health and disease and develop better treatments for a range of disorders.”

Dr. Sankaran, a hematologist/oncologist, is the Jan Ellen Paradise, MD, Professor of Pediatrics at Harvard Medical School. He is in the Division of Hematology/Oncology at Boston Children's Hospital and is an attending physician in the Dana-Farber/ Boston Children's Cancer and Blood Disorders Center, as well as an associate member of the Broad Institute of MIT and Harvard. Dr. Sankaran was recently selected as a Howard Hughes Medical Institute Investigator.

A jury of distinguished biomedical research leaders selected Dr. Sankaran as the 2024 Trailblazer Prize recipient. Michael J. Welsh, MD, PhD, Director, Pappajohn Biomedical Institute, University of Iowa, chaired the Trailblazer Prize jury, which also included Howard Y. Chang, MD, PhD; John I. Gallin, MD; Helen H. Hobbs, MD; Timothy J. Ley, MD; Steven M. Paul, MD; and Christine E. Seidman, MD.

The 2024 Trailblazer Prize includes an honorarium for Dr. Sankaran, made possible by a generous donation from John I. Gallin, MD, and Elaine Gallin, PhD, to the FNIH. The prize will be awarded at the 12th Annual FNIH Awards Ceremony on the evening of October 29, 2024, in Washington, D.C. The 2024 Lurie Prize in Biomedical Sciences and the Charles A. Sanders, MD, Partnership Award also will be presented during the Awards Ceremony.

Dr. Sankaran will share his research at the 2025 AAP/ASCI/APSA Joint Meeting in Chicago occurring April 25-27. This combined annual gathering of the Association of American Physicians, the American Society for Clinical Investigation, and the American Physician Scientists Association features presentations of high-level discoveries from distinguished researchers.

The FNIH gratefully acknowledges its Annual Awards Ceremony Premier Sponsors, Fred and Donna Seigel, and our Visionary Sponsors, Sherry and Alan Leventhal, Paul and Sandra Montrone, Dr. Gilbert S. Omenn and Ms. Martha A. Darling, and Steven and Jann Paul, MD.

For more information about the Trailblazer Prize, visit fnih.org/TrailblazerPrize.

About the Foundation for the National Institutes of Health

The Foundation for the National Institutes of Health (FNIH) builds public-private partnerships that connect leading biomedical scientists at the National Institutes of Health (NIH) with their counterparts in life sciences companies, academia, patient organizations, foundations, and regulatory agencies (including the Food and Drug Administration and European Medicines Agency). Through team science, the FNIH solves complex health challenges and accelerates breakthroughs for patients, regardless of who they are or what health threats they face. The FNIH contributes to the development of new therapies, diagnostics, and potential cures; advances global health and equity in care; and celebrates and helps train the next generations of scientists. Established by Congress in 1990 to support the mission of the NIH, the FNIH is a not-for-profit 501(c)(3) charitable organization. For more information about the FNIH, please visit fnih.org.

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Kathy Scarbeck FNIH 301-827-6648 kscarbeck@fnih.org