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How sickle cell became the first CRISPR’d disease
Nearly a decade ago, consultants delivered to Rodger Novak a kind of Sears catalog of human malady: 200 pages, listing dozens of different diseases, each annotated with — from a business standpoint — their best and worst attributes. It was supposed to help Novak, then the chief executive of CRISPR Therapeutics, navigate a pressing quandary. His company was founded to commercialize the new revolutionary gene-editing tool CRISPR-Cas9, which promised to cure numerous genetic diseases. But which should they target first?
The result today is one of the more welcome twists of the CRISPR story. Novak dismissed all the consultants’ recommendations and picked sickle cell disease. Now, these patients who have long faced discrimination in medical settings, stand poised to be the first to benefit from a groundbreaking technology. Any day, the FDA is expected to approve exa-cel, the treatment built by CRISPR Therapeutics and its partner Vertex Pharmaceuticals.
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