What gene did Vijay Sankaran's team identify as key to sickle cell disease?

The team identified the BCL11A gene as a key regulator of fetal hemoglobin production.

When was the first-ever gene-editing medication for sickle cell disease approved?

The FDA approved the gene-editing medication Casgevy in 2023.

How did Vijay Sankaran's experience with a speech impediment as a child shape his outlook?

Sankaran says his childhood speech therapy experience helped him face challenges head-on and believe he can overcome them.

Home / Health / Harvard Scientist's Chance Encounter Leads to Lifesaving Gene Therapy

Harvard Scientist's Chance Encounter Leads to Lifesaving Gene Therapy

17 Nov

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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.