After decades of neglect, stigma and underfunding, sickle cell is getting the equivalent of the red carpet treatment in science. It’s the target of a competitive biotech race, with scientists and companies using a crop of cutting-edge tools to try to cure the debilitating illness.
The first gene therapies for sickle cell, including one based on the buzzy, Nobel Prize-winning technique called CRISPR, will be reviewed by regulators this year, and companies are preparing to launch the medicines if they get the green light. That puts the country at the cusp of two frontiers: a new era in treating a tragically overlooked disease, and the beginning of what could be a CRISPR revolution in medicine.
It’s a dramatic about-face for sickle cell patients, who have often felt abandoned by the medical system. The rare disease afflicts about 100,000 people in the United States, most of them Black. Racism at both the institutional and interpersonal level has stymied funding and alienated patients.
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“The hope,” said [Sick Cells president Ashley] Valentine, “is that if the feds and governments and society can figure this out with sickle cell, they can figure this out with other diseases.”
