Stuart Orkin receives the third Elaine Redding Brinster Prize for development of sickle cell disease therapy

Reviewed by Danielle Ellis, B.Sc.Sep 15 2023

For his work discovering the basis for hemoglobin gene switching and applying those insights to develop a therapy for sickle cell disease and other blood diseases, the Institute for Regenerative Medicine at the University of Pennsylvania awarded Stuart Orkin, MD the third Elaine Redding Brinster Prize in Science or Medicine.

Orkin's research advanced the understanding of how the fetal hemoglobin gene-; the main oxygen carrier protein in the human fetus-;is silenced in adults. He also developed a therapy that re-activates the fetal gene for adult hemoglobin gene defects, which cause red blood cell diseases.

Dr. Orkin has beautifully illustrated how a career of basic science investigation into the mechanisms for gene regulation can be applied, in one's own laboratory, to a method for combating devastating human diseases. Notably, his discovery of unexpected details in how the fetal hemoglobin gene is regulated suggested insights for a therapy, for which he availed of the latest gene editing technologies to develop a specific clinical application for sickle cell disease. We are thrilled that Dr. Orkin is the third awardee of the Elaine Redding Brinster Prize."

Ken Zaret, PhD, director of Penn's Institute for Regenerative Medicine and the Joseph Leidy Professor of Cell and Developmental Biology in the Perelman School of Medicine

The prize, supported by an endowment from the children of Elaine Redding Brinster, is awarded annually to a researcher whose singular discovery has made a unique impact on biomedicine. Each winner receives $100,000, a commemorative medal, and an invitation to present a ceremonial lecture at the University of Pennsylvania.

Orkin will accept the prize on March 13, 2024, as part of the day-long Ralph L. Brinster Symposium at Penn's Philadelphia campus. The symposium will feature eminent speakers from across the biomedical sciences, including Titia de Lange, PhD, of Rockefeller University; Carla Shatz, PhD, of Stanford University; Alejandro Sànchez Alvarado, PhD, of the Stowers Institute for Medical Research; and Marianne Bronner, PhD, of the California Institute of Technology.

"I am very honored, and humbled, by recognition with the Brinster Prize. I hope that work of my laboratory will inspire others to pursue a career of fundamental discovery for the benefit of patients," said Orkin, the David G. Nathan Distinguished Professor of Pediatrics, Harvard Medical School, and investigator with Howard Hughes Medical Institute.

Orkin has been honored with several prestigious awards, including the Canada Gairdner International Award, the Gruber Foundation Prize in Genetics, the King Faisal Prize in Medicine, the Kovaleno Medal of the National Academy of Sciences, and the Harrington Discovery Institute Prize for Innovation in Medicine. Orkin is also a member of the U.S. National Academy of Medicine and National Academy of Sciences.

Previous recipients of the Elaine Redding Brinster Prize include molecular biologist C. David Allis, PhD, and neurogeneticist Huda Zoghbi, MD.

The Penn Institute for Regenerative Medicine is dedicated to researching cells and tissues with an eye toward turning the knowledge gained into new diagnostic and therapeutic techniques and tools. A member of the International Society for Stem Cell Research's (ISSCR) Circle of Stem Cell Institute and Center Directors, the institute features faculty from five schools across the University of Pennsylvania and includes representation from Children's Hospital of Philadelphia and the Wistar Institute.