In late 2023, the Food and Drug Administration (FDA) made a landmark decision by approving two gene therapies for sickle cell disease, an inherited red blood cell disorder affecting millions globally. Notably, one of these therapies became the first FDA-approved treatment to use the CRISPR gene-editing tool, marking the culmination of decades of scientific discoveries.
Vijay Sankaran, PhD ’09, MD ’10, the Jan Ellen Paradise, MD Professor of Pediatrics at Boston Children’s Hospital, played a pivotal role in one such discovery back in 2008, when he was a student in the Harvard/MIT MD-PhD Program.
“The program’s exposure to cutting-edge human genetics through exceptional teachers like Joel Hirschhorn (AB ’86, MD ’95, PhD ’95) and David Altshuler (MD ’94, PhD ’94)—both MD-PhD Program alumni—directly inspired our use of genetic studies to decipher fetal hemoglobin regulation, culminating in the discovery of BCL11A as a key regulator of fetal hemoglobin, which prevents red blood cells from sickling,” Sankaran says.
This dedication to pioneering research that impacts patients exemplifies the ethos of the Harvard/MIT MD-PhD Program, which has provided dual clinical and research training for over 50 years, preparing students to become leading physician-scientists. Sankaran highlights the program’s incredible community, which unites world-class faculty from Harvard Medical School and its affiliated hospitals, the Massachusetts Institute of Technology (MIT), and Harvard’s main campus with a diverse, creative, and innovative student body.
The donor’s extraordinary commitment to supporting MD-PhD students in the basic sciences empowers this next generation of physician-scientists to push the boundaries of discovery and innovation at the interface of science and medicine, driving advancements in patient care.