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How Erica Esrick and David A. Williams are using gene therapy to fight sickle cell disease

For developing a successful way to reengineer patients’ blood stem cells, Erica Esrick and David A. Williams are among Fast Company’s Most Creative People in Business for 2020

How Erica Esrick and David A. Williams are using gene therapy to fight sickle cell disease

Sickle cell disease is the most common inherited blood disorder in the U.S., affecting more than 100,000 Americans, including 1 in 365 African Americans. The disease affects hemoglobin, the molecule in red blood cells that carries oxygen throughout the body, causing the cells to take on a crescent, or sickle, shape. These deformed cells are rigid and break apart easily, which leads to anemia, organ damage, and bouts of severe pain. “Sickle cell is challenging because it’s such a protean disease,” says David A. Williams, senior vice president and chief scientific officer at Boston Children’s Hospital. The only effective treatment has been a bone marrow transplant, most often from a healthy sibling. But a novel gene therapy developed in Williams’s lab reengineers a patient’s blood stem cells to increase production of so-called fetal hemoglobin, which is optimized for the low-oxygen environment of the womb and has potent anti-sickling characteristics. The human body stops making fetal hemoglobin after birth, but by “silencing” a certain gene, production can be restarted, creating healthier red blood cells. In a clinical trial launched in late 2018 and led by physician Erica Esrick, patients’ blood stem cells are modified outside the body and infused back into their bloodstream. In five patients with results reports so far, the infusion increased fetal hemoglobin production—and reduced symptoms. “This is an exciting time,” Esrick says. The FDA green-lighted an expansion of the trials last fall.

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