Home / Health / Harvard Scientist's Chance Encounter Leads to Lifesaving Gene Therapy
Harvard Scientist's Chance Encounter Leads to Lifesaving Gene Therapy
17 Nov
Summary
- Vijay Sankaran, a Harvard medical student, pivoted his research to focus on sickle cell disease
- His team identified a key gene, BCL11A, that regulates fetal hemoglobin production
- In 2023, the FDA approved the first-ever gene-editing medication for sickle cell disease
In 2025, Vijay Sankaran, a pediatric hematologist and oncologist at Boston Children's Hospital and professor at Harvard Medical School, is continuing his pioneering research on blood disorders. Sankaran's journey began over a decade ago when, as a first-year medical student at Harvard, he met a sickle cell patient whose condition was greatly improved by a treatment that stimulated fetal hemoglobin production.
Inspired by this chance encounter, Sankaran decided to shift his focus from stem cells and structural biology to studying fetal hemoglobin. Despite initial setbacks, his team was one of the first to examine the genetics of fetal hemoglobin and in 2008 identified the key BCL11A gene that regulates its production. This breakthrough led to the development of a sickle cell gene therapy.
In 2023, the U.S. Food and Drug Administration approved Casgevy, a commercial treatment that edits the BCL11A gene to restart sickle cell patients' fetal hemoglobin production. Casgevy is the first-ever gene-editing medication, a testament to Sankaran's perseverance and his patient-centered approach to research.
As Sankaran continues to lead his lab's efforts to uncover the genetic underpinnings of blood disorders, he remains committed to designing new treatments for conditions like anemia and leukemia. His work has already identified thousands of gene variants that play a role in disease, and he encourages his students to take risks and pursue their passions, just as his mentor did for him.
