CNBC December 7, 2023
Key Points
– The U.S. Food and Drug Administration is expected to approve exa-cel gene-editing treatment for sickle cell disease.
– Exa-cel would be the first approved medicine in the U.S. to use CRISPR gene-editing technology.
– Vertex Pharmaceuticals and CRISPR Therapeutics co-developed the treatment, which could cost around $2 million per patient.
At age 19, Joe Tsogbe underwent his first hip replacement. In his 20s, he averaged about nine hospitalizations a year. By his 30s, that rose to more than a dozen.
All the result of sickle cell disease, an inherited blood disorder where a genetic mutation causes normally full-moon shaped red blood cells to form into half moons and get stuck inside blood vessels, restricting blood flow...
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